
Vertex Pharmaceuticals Inc
VRTXHealthcare|Biotechnology|USA
$434.30
-4.41 (-1.01%)
DCF (FCF)
$183.42
Tangible Book
$67.54
Graham Number
$167.26
Earnings Power
$127.09
Clinical Trials (283)
| Trial | Conditions | Phase | Status | Enrollment | Design | Drug |
|---|---|---|---|---|---|---|
| NCT05329649 Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) | Sickle Cell Disease, Hydroxyurea Failure | P3 | Active | 13 | Open-label | CTX001 |
| NCT06460506 Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older | Cystic Fibrosis | P3 | Active | 50 | Open-label | ELX/TEZ/IVA, IVA |
| NCT05312879 Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney Disease | Proteinuric Kidney Disease | P2P3 | Recruiting | 466 | RCT, Double-blind | VX-147, Placebo |
| NCT07231419 Evaluation of Efficacy and Safety of Suzetrigine (SUZ) for Pain Associated With Diabetic Peripheral Neuropathy | Diabetic Peripheral Neuropathic Pain | P3 | Recruiting | 734 | RCT, Double-blind | Suzetrigine, Placebo (matched to SUZ) |
| NCT05356195 Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) | Beta-Thalassemia, Thalassemia | P3 | Active | 16 | Open-label | CTX001 |
| NCT04786262 A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 Diabetes | Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness | P3 | Recruiting | 52 | Open-label | VX-880 |
| NCT05331183 Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del | Cystic Fibrosis | P3 | Active | 297 | Open-label | ELX/TEZ/IVA, IVA |
| NCT06628908 Evaluation of Efficacy and Safety of Suzetrigine for Pain Associated With Diabetic Peripheral Neuropathy | Diabetic Peripheral Neuropathic Pain | P3 | Recruiting | 1,100 | RCT, Double-blind | Suzetrigine, Placebo (matched to SUZ), Pregabalin, Placebo (matched to Pregabalin) |
| NCT06564142 Evaluation of Efficacy of Povetacicept in Adults With Immunoglobulin A Nephropathy (IgAN) | Immunoglobulin A Nephropathy | P3 | Active | 605 | RCT, Double-blind | Povetacicept, Placebo (matched to Povetacicept) |
| NCT05477563 Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | Beta-Thalassemia, Thalassemia | P3 | Recruiting | 26 | Open-label | CTX001 |
| NCT06696443 Evaluation of the Long-term Safety and Effectiveness of Suzetrigine (SUZ) in Participants With Painful Diabetic Peripheral Neuropathy (DPN) | Diabetic Peripheral Neuropathic Pain | P3 | Active | 455 | Open-label | Suzetrigine |
| NCT07204275 A Study to Evaluate Efficacy, Safety, and Tolerability of Povetacicept in Participants With Primary Membranous Nephropathy (pMN) | Primary Membranous Nephropathy | P2P3 | Recruiting | 176 | RCT, Double-blind | Povetacicept, Tacrolimus |
| NCT05444257 A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy | Cystic Fibrosis | P3 | Active | 822 | Open-label | VX-121/TEZ/D-IVA |
| NCT05422222 Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age | Cystic Fibrosis | P3 | Recruiting | 210 | Open-label | VX-121/TEZ/D-IVA |
| NCT06832410 An Efficacy, Safety, and Tolerability Study of VX-880 in Participants With Type 1 Diabetes With a Kidney Transplant | Type 1 Diabetes, Kidney Transplant | P3 | Recruiting | 10 | Open-label | Biological/Vaccine |
| NCT05668741 A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF) | Cystic Fibrosis | P1P2 | Recruiting | 39 | Open-label | VX-522 mRNA therapy, IVA |
| NCT06619860 Evaluation of Efficacy and Safety of VX-993 for Pain Associated With Diabetic Peripheral Neuropathy | Diabetic Peripheral Neuropathic Pain | P2 | Recruiting | 300 | RCT, Double-blind | VX-993, Pregabalin, Placebo (matched to pregabalin), Placebo (matched to VX-993) |
| NCT07161037 Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW) | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | P2 | Recruiting | 24 | Open-label | VX-407 |
| NCT05791201 A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes | Type 1 Diabetes | P1P2 | Active | 7 | Open-label | VX-264 |
| NCT06794996 Inaxaplin in Participants With Proteinuric APOL1 Mediated Kidney Disease With or Without Comorbidities | Proteinuric Kidney Disease | P2 | Recruiting | 45 | Open-label | Inaxaplin |
| NCT06185764 A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) | Myotonic Dystrophy Type 1 (DM1) | P1P2 | Recruiting | 44 | RCT, Double-blind | VX-670, Placebo |
| NCT07437105 Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants | Cystic Fibrosis | P1 | Recruiting | 128 | RCT, Double-blind | VX-272, Placebo |
| NCT06154447 Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis | Cystic Fibrosis | P1 | Recruiting | 255 | RCT, Double-blind | VX-828, Placebo, Itraconazole, Midazolam, Tezacaftor, VX-118, Placebo, Deutivacaftor, VX-828 |
| NCT07283770 Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy Participants | Cystic Fibrosis | P1 | Recruiting | 128 | RCT, Double-blind | VX-581, Placebo |
| NCT07378865 Evaluation of the Excretion of Suzetrigine Into Breast Milk in Healthy Lactating Female Participants | Pain | P1 | Recruiting | 12 | Open-label | Suzetrigine |
| NCT06747572 Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Active | 401 | — | — | |
| NCT06887959 A Single-arm Study Evaluating the Effectiveness and Safety of Suzetrigine (SUZ) for Acute Pain After Selected Surgeries | Acute Pain | P4 | Completed | 47 | Open-label | Suzetrigine |
| NCT01467492 Telaprevir, Peg-IFN-alfa-2a, and RBV in Treatment-Experienced Black/African American and Non-Black/African American Subjects With Genotype 1 Chronic Hepatitis C | Hepatitis C | P4 | Terminated | 121 | Open-label | Telaprevir, Ribavirin, Pegylated Interferon Alfa-2a |
| NCT07463430 Suzetrigine for Non-Mastectomy Breast Surgery | Pain, Acute Postoperative, Breast Disease | P4 | Not Yet Recruiting | 120 | Open-label | suzetrigine |
| NCT02823470 A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del CFTR Mutation | Cystic Fibrosis | P4 | Terminated | 24 | RCT, Open-label | LUM/IVA |
| NCT06887972 A Single-Arm Study Evaluating the Effectiveness and Safety of Suzetrigine (SUZ) for Acute Pain After Aesthetic or Reconstructive Surgeries | Acute Pain | P4 | Completed | 100 | Open-label | Suzetrigine |
| NCT02875366 A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation | Cystic Fibrosis | P4 | Completed | 70 | RCT, Double-blind | LUM/IVA, Placebo |
| NCT02934698 An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations | Cystic Fibrosis | P3 | Completed | 2 | Open-label | Ivacaftor |
| NCT01117012 Rollover Study of VX-770 in Cystic Fibrosis Subjects | Cystic Fibrosis | P3 | Completed | 192 | Open-label | Ivacaftor |
| NCT05153317 Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older | Cystic Fibrosis | P3 | Completed | 71 | Open-label | ELX/TEZ/IVA, IVA |
| NCT01054573 VX-950-TiDP24-C219: A Roll Over Trial for Patients in the Control Group of the C216 Study Who Received Telaprevir Placebo | Hepatitis C, Chronic | P3 | Completed | 90 | Open-label | Telaprevir, pegylated interferon (Peg-IFN) alfa-2a, ribavirin (RBV) |
| NCT03125395 A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation | Cystic Fibrosis | P3 | Completed | 57 | Open-label | LUM/IVA |
| NCT00758043 A Study Evaluating 24-Week and 48-Week Telaprevir-Based Regimen in Treatment Naïve Subjects With Genotype 1 Chronic Hepatitis C Who Achieve an Extended Rapid Viral Response | Hepatitis C | P3 | Completed | 540 | RCT, Open-label | telaprevir, ribavirin, peginterferon alfa-2a |
| NCT02797132 Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del | Cystic Fibrosis | P3 | Completed | 62 | Open-label | LUM/IVA |
| NCT03150719 A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF) | Cystic Fibrosis | P3 | Completed | 98 | RCT, Double-blind | Tezacaftor/Ivacaftor, Ivacaftor, Placebo, Placebo |
| NCT02544451 Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor | Cystic Fibrosis | P3 | Completed | 246 | Open-label | LUM/IVA, LUM/IVA |
| NCT05844449 Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older | Cystic Fibrosis | P3 | By Invite | 174 | Open-label | VNZ/TEZ/D-IVA |
| NCT02390219 Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease | Cystic Fibrosis, Advanced Lung Disease | P3 | Completed | 46 | Open-label | Lumacaftor, Ivacaftor |
| NCT03601637 Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del | Cystic Fibrosis | P3 | Completed | 61 | Open-label | LUM, IVA |
| NCT02347657 A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor | Cystic Fibrosis | P3 | Completed | 510 | RCT, Double-blind | VX-661 Plus Ivacaftor Combination, Ivacaftor, VX-661 Plus Ivacaftor Combination Placebo, Ivacaftor placebo |
| NCT04537793 Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years | Cystic Fibrosis | P3 | Completed | 83 | Open-label | ELX/TEZ/IVA, IVA |
| NCT04353817 A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes | Cystic Fibrosis | P3 | Completed | 121 | RCT, Double-blind | ELX/TEZ/IVA, IVA |
| NCT05558410 Evaluation of Efficacy and Safety of Suzetrigine for Acute Pain After an Abdominoplasty | Acute Pain | P3 | Completed | 1,118 | RCT, Double-blind | Suzetrigine (SUZ), HB/APAP, Placebo (matched to SUZ), Placebo (matched to HB/APAP) |
| NCT01946412 Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation | Cystic Fibrosis | P3 | Completed | 33 | Open-label | Ivacaftor |
| NCT05661734 A Single-arm Study to Evaluate Safety and Effectiveness of VX-548 for Acute Pain | Pain | P3 | Completed | 258 | Open-label | Suzetrigine |
| NCT04362761 A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy | Cystic Fibrosis | P3 | Completed | 172 | Open-label | ELX/TEZ/IVA, IVA |
| NCT01807949 A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation | Cystic Fibrosis, Homozygous for the F508del CFTR Mutation | P3 | Completed | 563 | RCT, Double-blind | Placebo, Lumacaftor Plus Ivacaftor Combination, Ivacaftor |
| NCT03447262 A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy | Cystic Fibrosis | P3 | Terminated | 484 | Open-label | VX-659/TEZ/IVA, IVA |
| NCT00781274 Efficacy and Safety of MP-424, Peginterferon Alfa-2b and Ribavirin in Chronic Hepatitis C Who Have Not Achieved an Undetectable HCV RNA Level With Previous Interferon Based Therapy | Hepatitis C | P3 | Completed | 32 | Open-label | MP-424, Ribavirin, Peginterferon Alfa-2b |
| NCT01890772 Vitamin D + Telaprevir + Peginterferon + Ribavirin for of Hepatitis C Genotype 1 | Hepatitis C | P2P3 | Withdrawn | — | RCT, Open-label | Telaprevir, Peginterferon alfa-2a, Ribavirin |
| NCT01614457 Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT) | Cystic Fibrosis | P3 | Completed | 70 | RCT, Double-blind | Ivacaftor, Placebo |
| NCT03559062 A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis | Cystic Fibrosis | P3 | Completed | 67 | RCT, Double-blind | TEZ/IVA, IVA, Placebo, Placebo |
| NCT02565914 A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation | Cystic Fibrosis | P3 | Completed | 1,131 | Open-label | TEZ/IVA, IVA |
| NCT00627926 A Phase 3 Study of Telaprevir in Combination With Pegasys® and Copegus® in Treatment-Naive Subjects With Genotype 1 Hepatitis C Virus (HCV) | Hepatitis C | P3 | Completed | 1,095 | RCT, Double-blind | Pegylated Interferon Alfa 2a, Telaprevir, Ribavirin |
| NCT00780910 Efficacy and Safety of MP-424, Peginterferon Alfa-2b, and Ribavirin in Patients With Chronic Hepatitis C Who Relapsed After Previous Interferon Based Therapy | Chronic Hepatitis C | P3 | Completed | 109 | Open-label | MP-424, Ribavirin, Peginterferon Alfa-2b |
| NCT03068312 A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation | Cystic Fibrosis | P3 | Completed | 38 | RCT, Double-blind | Ivacaftor, Placebo |
| NCT00909727 Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation | Cystic Fibrosis | P3 | Completed | 52 | RCT, Double-blind | Ivacaftor, Placebo |
| NCT01830985 A Phase 2/3 Open-label Extension Study to Evaluate Long-Term Safety and Efficacy With VX-509 in Subjects With Rheumatoid Arthritis | Rheumatoid Arthritis | P2P3 | Completed | 39 | Open-label | VX-509 |
| NCT05033080 A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF) | Cystic Fibrosis | P3 | Completed | 435 | RCT, Double-blind | VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, Placebo (matched to VX-121/TEZ/D-IVA), Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA) |
| NCT02516410 A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation | Cystic Fibrosis | P3 | Completed | 168 | RCT, Double-blind | VX-661 plus ivacaftor combination, Ivacaftor, Placebo (matched to VX-661 plus ivacaftor combination), Placebo (matched to ivacaftor) |
| NCT04043806 A Study Evaluating the Long-term Safety of VX-445 Combination Therapy | Cystic Fibrosis | P3 | Completed | 458 | Open-label | ELX/TEZ/IVA, IVA |
| NCT01821963 Telaprevir Plus Standard of Care (SOC) in HCV Associated Hepatocellular Carcinoma (HCC) | Infection | P3 | Terminated | 1 | Open-label | Pegylated Interferon Alfa 2a, Ribavirin, Telaprevir |
| NCT03447249 A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) | Cystic Fibrosis | P3 | Completed | 385 | RCT, Double-blind | VX-659/TEZ/IVA, IVA, Placebo |
| NCT02669849 Study to Assess the Efficacy and Safety of VX-210 in Subjects With Acute Traumatic Cervical Spinal Cord Injury | Cervical Spinal Cord Injury | P2P3 | Terminated | 70 | RCT, Double-blind | VX-210, Placebo |
| NCT01707290 Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation | Cystic Fibrosis | P3 | Completed | 125 | Open-label | Ivacaftor |
| NCT03633526 Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age | Cystic Fibrosis | P3 | Terminated | 18 | Open-label | VX-659/TEZ/IVA, IVA |
| NCT02412111 A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor | Cystic Fibrosis | P3 | Completed | 156 | RCT, Double-blind | Ivacaftor, Tezacaftor/Ivacaftor |
| NCT04183790 Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older | Cystic Fibrosis | P3 | Completed | 64 | Open-label | ELX/TEZ/IVA, IVA |
| NCT03525574 A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy | Cystic Fibrosis | P3 | Completed | 507 | Open-label | ELX/TEZ/IVA, IVA |
| NCT01459913 Efficacy of a 12-Week Regimen of Telaprevir, Pegylated Interferon, and Ribavirin in Treatment-Naive and Prior Relapser Subjects With Interleukin28B (IL28B) CC Genotype | Hepatitis C, Chronic | P3 | Terminated | 239 | RCT, Open-label | Telaprevir, Pegylated Interferon Alfa-2a, Ribavirin |
| NCT02514473 A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation | Cystic Fibrosis | P3 | Completed | 206 | RCT, Double-blind | VX-809, Placebo, VX-770 |
| NCT04105972 A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del | Cystic Fibrosis | P3 | Completed | 176 | RCT, Double-blind | ELX/TEZ/IVA, TEZ/IVA, IVA |
| NCT03537651 A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation | Cystic Fibrosis | P3 | Completed | 130 | Open-label | TEZ/IVA, IVA |
| NCT05111145 A Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Participants With Cystic Fibrosis (CF) | Cystic Fibrosis | P3 | Completed | 86 | Open-label | ELX/TEZ/IVA, IVA |
| NCT04058353 A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) | Cystic Fibrosis | P3 | Completed | 271 | RCT, Double-blind | ELX/TEZ/IVA, IVA, TEZ/IVA |
| NCT03525548 A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F) | Cystic Fibrosis | P3 | Completed | 113 | RCT, Double-blind | VX-445/TEZ/IVA, TEZ/IVA, IVA, Placebo, Placebo |
| NCT03277196 A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation | Cystic Fibrosis | P3 | Completed | 86 | Open-label | IVA |
| NCT04969224 A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF) | Cystic Fibrosis | P3 | Completed | 82 | Open-label | ELX/TEZ/IVA, IVA |
| NCT01241760 VX-950-C211 - A Dosing Regimen Study (Twice Daily Versus Every 8 Hours) of Telaprevir in Treatment-naïve Participants With Genotype 1 Chronic Hepatitis C Virus Infection | Genotype 1 Chronic Hepatitis C, Treatment Naive | P3 | Completed | 744 | RCT, Open-label | Ribavirin, Telaprevir, Pegylated interferon alfa-2a, Telaprevir |
| NCT04599465 A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF) | Cystic Fibrosis | P3 | Completed | 69 | Open-label | ELX/TEZ/IVA, IVA |
| NCT04235140 Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation | Cystic Fibrosis | P3 | Completed | 52 | Open-label | LUM/IVA |
| NCT04545515 A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes | Cystic Fibrosis | P3 | Completed | 120 | Open-label | ELX/TEZ/IVA, IVA |
| NCT03460990 A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F) | Cystic Fibrosis | P3 | Completed | 116 | RCT, Double-blind | VX-659/TEZ/IVA, TEZ/IVA, IVA, Placebo, Placebo |
| NCT02392234 A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation | Cystic Fibrosis | P3 | Completed | 248 | RCT, Double-blind | VX-661/Ivacaftor, Ivacaftor, Placebo matched to VX-661/ ivacaftor, Placebo matched to Ivacaftor |
| NCT05882357 Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age | Cystic Fibrosis | P3 | Completed | 70 | Open-label | ELX/TEZ/IVA, IVA |
| NCT01807923 A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation | Cystic Fibrosis, Homozygous for the F508del CFTR Mutation | P3 | Completed | 559 | RCT, Double-blind | Lumacaftor Plus Ivacaftor Combination, Ivacaftor, Placebo |
| NCT03745287 A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease | Sickle Cell Disease, Hematological Diseases | P2P3 | Completed | 63 | Open-label | CTX001 |
| NCT01897233 Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation | Cystic Fibrosis | P3 | Completed | 62 | Open-label | Lumacaftor, Ivacaftor |
| NCT01614470 Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation | Cystic Fibrosis | P3 | Completed | 39 | RCT, Double-blind | Ivacaftor, Placebo |
| NCT01705145 Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation | Cystic Fibrosis | P3 | Completed | 35 | Open-label | Ivacaftor |
| NCT00780416 Efficacy and Safety of MP-424/Peginterferon Alfa-2b/Ribavirin Combination in Treatment-Naïve Patients With Chronic Hepatitis C | Hepatitis C | P3 | Completed | 189 | RCT, Open-label | MP-424, Ribavirin, Peginterferon Alfa-2b, Ribavirin, Peginterferon Alfa-2b |
| NCT02953314 A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF) | Cystic Fibrosis | P3 | Completed | 83 | Open-label | TEZ, TEZ/IVA, IVA |
| NCT05076149 A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation | Cystic Fibrosis | P3 | Completed | 597 | RCT, Double-blind | VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, Placebo (matched to VX-121/TEZ/D-IVA), Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA) |
| NCT01467479 A Study to Treat Subjects With Telaprevir, Ribavirin, and Peginterferon Who Are Coinfected With HIV and Hepatitis C Virus (HCV) | Hepatitis C | P3 | Terminated | 185 | Open-label | Telaprevir, Ribavirin, Pegylated Interferon Alfa-2a, Highly Active Antiretroviral Therapy (HAART) |
| NCT01931839 A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis | Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation | P3 | Completed | 1,164 | RCT, Double-blind | Lumacaftor Plus Ivacaftor Combination, Ivacaftor |
| NCT04058366 Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy | Cystic Fibrosis | P3 | Completed | 251 | Open-label | ELX/TEZ/IVA, IVA |
| NCT05951205 Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype | Sickle Cell Disease | P3 | Withdrawn | — | Open-label | Exa-cel |
| NCT03691779 Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age | Cystic Fibrosis | P3 | Completed | 71 | Open-label | ELX/TEZ/IVA, IVA |
| NCT02742519 A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation | Cystic Fibrosis | P3 | Terminated | 14 | RCT, Double-blind | ivacaftor, Placebo |
| NCT05553366 Evaluation of Efficacy and Safety of VX-548 for Acute Pain After a Bunionectomy | Acute Pain | P3 | Completed | 1,075 | RCT, Double-blind | SUZ, HB/APAP, Placebo (matched to SUZ), Placebo (matched to HB/APAP) |
| NCT03525444 A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) | Cystic Fibrosis | P3 | Completed | 405 | RCT, Double-blind | VX-445/TEZ/IVA, IVA, Placebo |
| NCT00909532 Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation | Cystic Fibrosis | P3 | Completed | 167 | RCT, Double-blind | Ivacaftor, Placebo |
| NCT05274269 Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation | Cystic Fibrosis | P3 | Completed | 307 | RCT, Double-blind | ELX/TEZ/IVA, IVA |
| NCT02725567 A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation | Cystic Fibrosis | P3 | Completed | 57 | Open-label | IVA |
| NCT03655678 A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia | Beta-Thalassemia, Thalassemia | P2P3 | Completed | 59 | Open-label | CTX001 |
| NCT04208529 A Long-term Follow-up Study in Participants Who Received CTX001 | Beta-Thalassemia, Thalassemia | P3 | By Invite | 160 | Open-label | CTX001 |
| NCT00535847 A Rollover Study for Subjects Participating in the Control Arm of Study VX06-950-106, VX05-950-104 and VX05-950-104EU Whose Plasma Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) Levels Did Not Respond to Therapy | Hepatitis C | P2 | Completed | 117 | Open-label | Telaprevir, Ribavirin, Pegylated interferon alfa 2a |
| NCT01790100 A Phase 2a, Dose-Ranging Study to Evaluate the Safety, Pharmacokinetics and Efficacy of VX-135 With Ribavirin in Treatment-Naïve Subjects With Chronic Hepatitis C | Chronic Hepatitis C | P2 | Completed | 20 | RCT, Double-blind | VX-135, Ribavirin |
| NCT01262352 Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation | Cystic Fibrosis | P2 | Completed | 21 | RCT, Double-blind | Ivacaftor, Placebo |
| NCT01467505 An Open Label Study of the Effect of Telaprevir in Combination With Ribavirin and Peginterferon on HCV Infection in Stable Liver Transplant Patients | Hepatitis C | P2 | Terminated | 61 | Open-label | Telaprevir, Ribavirin, Pegylated Interferon Alfa-2a, Immunosuppressant Regimen |
| NCT05660538 Evaluation of Efficacy and Safety of VX-548 for Painful Diabetic Peripheral Neuropathy (DPN) | Diabetic Peripheral Neuropathy | P2 | Completed | 194 | RCT, Double-blind | Suzetrigine, Pregabalin, Placebo (matched to SUZ), Placebo (matched to pregabalin) |
| NCT03206749 A Study to Evaluate Efficacy and Safety of VX-150 in Subjects With Acute Pain Following Bunionectomy | Acute Pain | P2 | Completed | 243 | RCT, Double-blind | VX-150, HB/APAP, Placebo |
| NCT00457821 Safety Study of Ivacaftor in Subjects With Cystic Fibrosis | Cystic Fibrosis | P2 | Completed | 39 | RCT, Double-blind | Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg, Ivacaftor 150 mg or 250 mg, Placebo |
| NCT00983853 Safety and Efficacy of Telaprevir in Combination With Peginterferon Alfa-2a and Ribavirin in Subjects Co-Infected With Hepatitis C Virus (HCV) and HIV | Hepatitis C, HIV Infections | P2 | Completed | 62 | RCT, Double-blind | telaprevir or matching placebo, telaprevir or matching placebo, peginterferon alfa-2a, ribavirin (fixed dose), ribavirin (weight-based dose) |
| NCT01161537 Study of the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis and the G551D Mutation | Cystic Fibrosis | P2 | Completed | 13 | Single-blind | VX-770, Placebo |
| NCT01994486 Open-Label Safety Study of Telaprevir and Sofosbuvir in Chronic Hepatitis C Genotype 1 | Hepatitis C, Chronic | P2 | Completed | 20 | Open-label | Telaprevir and Sofosbuvir |
| NCT03768089 Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis | Cystic Fibrosis | P1P2 | Completed | 115 | RCT, Double-blind | Placebo (matched to VX-121 suspension), VX-121 (Suspension), TEZ/IVA, IVA, Placebo (matched to TEZ/IVA), Placebo (matched to IVA), VX-121 (Tablet), Placebo (matched to VX-121 tablet) |
| NCT01581138 VX-222 + Telaprevir + Ribavirin for 12 or 16 Weeks in Treatment-Naive Subjects With Genotype 1a Hepatitis C | Chronic Hepatitis C Virus | P2 | Completed | 64 | RCT, Open-label | VX-222, telaprevir, ribavirin |
| NCT00336479 Phase 2 Study of VX-950, Pegasys®, and Copegus® in Hepatitis C | Chronic Hepatitis C | P2 | Completed | 263 | RCT, Double-blind | Telaprevir, Ribavirin, Pegylated Interferon Alfa 2a |
| NCT00420784 A Study of Telaprevir (VX-950), Pegasys and Copegus in Hepatitis C (PROVE3) | Hepatitis C | P2 | Completed | 465 | RCT, Double-blind | Telaprevir, Ribavirin, Pegylated Interferon Alfa 2a, Matching Placebo |
| NCT01501383 A Study to Evaluate the Efficacy and Safety of VX-765 in Subjects With Treatment-Resistant Partial Epilepsy | Epilepsy | P2 | Terminated | 55 | RCT, Double-blind | VX-765 Part A, Placebo, VX-765 Part B |
| NCT04006873 Gut Imaging for Function & Transit in Cystic Fibrosis Study 2 | Cystic Fibrosis | P2 | Completed | 12 | RCT, Double-blind | Tezacaftor/Ivacaftor + Ivacaftor, Placebo oral tablet |
| NCT02730208 A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation | Cystic Fibrosis | P2 | Completed | 41 | RCT, Double-blind | Tezacaftor/Ivacaftor, Ivacaftor, Placebo, Placebo |
| NCT07501702 A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia Gravis | Myasthenia Gravis, Generalized | P2 | Not Yet Recruiting | 30 | RCT, Double-blind | Povetacicept, Placebo |
| NCT01516918 A Study to Evaluate the Efficacy and Safety of Quadruple Therapy (VX-222, Telaprevir,Peginterferon-Alfa-2a, Ribavirin) in Subjects With Chronic Hepatitis C With Compensated Cirrhosis | Chronic Hepatitis C Virus | P2 | Completed | 103 | Open-label | VX-222, telaprevir, ribavirin, peginterferon-alfa-2a |
| NCT01701063 An Open-Label Study of the Effect of Telaprevir in Combination With Peginterferon Alfa-2b and Ribavirin in Pediatric Subjects Infected With Hepatitis C Virus | Hepatitis C | P1P2 | Terminated | 42 | Open-label | Telaprevir, Peginterferon alfa-2b, Ribavirin |
| NCT04167345 Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype | Alpha 1-Antitrypsin Deficiency | P2 | Terminated | 48 | RCT, Double-blind | VX-814, Placebo |
| NCT01048255 Study of VX-765 in Subjects With Treatment-resistant Partial Epilepsy | Partial Epilepsy | P2 | Completed | 60 | RCT, Double-blind | VX-765 |
| NCT00953706 Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation | Cystic Fibrosis | P2 | Terminated | 140 | RCT, Double-blind | Ivacaftor, Placebo |
| NCT06176196 Evaluation of Efficacy and Safety of VX-548 for Painful Lumbosacral Radiculopathy (PLSR) | Painful Lumbosacral Radiculopathy | P2 | Completed | 218 | RCT, Double-blind | VX-548, Placebo |
| NCT04066751 The Multicenter Topic Trial | Chronic Obstructive Pulmonary Disease | P2 | Withdrawn | — | RCT, Double-blind | Ivacaftor |
| NCT02070744 Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion | Cystic Fibrosis | P2 | Completed | 40 | RCT, Double-blind | VX-661, Ivacaftor, Placebo matched to VX-661, Placebo matched to Ivacaftor |
| NCT01685801 Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function | Cystic Fibrosis | P2 | Completed | 24 | RCT, Double-blind | Ivacaftor, Placebo-matched-to-ivacaftor tablet |
| NCT02709109 A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation | Cystic Fibrosis | P2 | Completed | 147 | RCT, Double-blind | VX-371 + HS, Hypertonic saline, Placebo, Orkambi, VX-371 |
| NCT00372385 Phase 2 Study of VX-950, Pegasys® With and Without Copegus® in Hepatitis C | Chronic Hepatitis C | P2 | Completed | 334 | RCT, Double-blind | Ribavirin, Pegylated Interferon Alfa 2a, Placebo, Telaprevir |
| NCT02660424 A Study of the Efficacy and Safety of VX-150 in Subjects With Osteoarthritis of the Knee | Osteoarthritis | P2 | Completed | 124 | RCT, Double-blind | VX-150, Placebo |
| NCT04474197 Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype | Alpha1-Antitrypsin Deficiency | P2 | Completed | 44 | RCT, Double-blind | VX-864, Placebo |
| NCT04977336 A Study Evaluating Efficacy and Safety of VX-548 for Acute Pain After a Bunionectomy | Acute Pain | P2 | Completed | 274 | RCT, Double-blind | VX-548, HB/APAP, Placebo (matched to VX-548), Placebo (matched to HB/APAP) |
| NCT01754935 A Magnetic Resonance Imaging Study and Arthroscopic Biopsy Substudy in Subjects With Active Rheumatoid Arthritis Receiving VX-509, an Oral JAK3 Inhibitor | Rheumatoid Arthritis | P2 | Completed | 43 | RCT, Double-blind | VX-509, VX-509 matching placebo |
| NCT00205465 Phase 2 Clinical Study in Psoriasis With Oral Investigational Drug VX-765 | Psoriasis | P2 | Completed | 64 | RCT, Double-blind | VX-765 |
| NCT06619847 A Study Evaluating Efficacy and Safety of VX-993 for Acute Pain After a Bunionectomy | Acute Pain | P2 | Completed | 367 | RCT, Double-blind | VX-993, HB/APAP, Placebo (matched to VX-993), Placebo (matched to HB/APAP) |
| NCT01225211 Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation | Cystic Fibrosis | P2 | Completed | 312 | RCT, Double-blind | Lumacaftor, Ivacaftor, Lumacaftor Placebo, Ivacaftor Placebo |
| NCT03061331 Lumacaftor/Ivacaftor Combination Therapy in Subjects With CF Who Have an A455E CFTR Mutation | Cystic Fibrosis | P2 | Completed | 20 | RCT, Double-blind | LUM/IVA, Placebo |
| NCT00395577 A Study in Rheumatoid Arthritis With an Investigational Oral p38 MAP Kinase Inhibitor VX-702 | Rheumatoid Arthritis | P2 | Completed | 120 | RCT, Double-blind | VX-702 |
| NCT03224351 A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis | Cystic Fibrosis | P2 | Completed | 124 | RCT, Double-blind | VX-659, TEZ/IVA, IVA, Placebo (matched to VX-659/TEZ/IVA), TEZ, VX-561, Placebo (matched to VX-659/TEZ/VX-561) |
| NCT04835441 ALPN-101 (Acazicolcept) in Systemic Lupus Erythematosus | Systemic Lupus Erythematosus | P2 | Completed | 76 | RCT, Double-blind | ALPN-101, Placebo |
| NCT02951195 A Study Evaluating the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis | Cystic Fibrosis | P2 | Completed | 80 | RCT, Double-blind | VX-152, TEZ/IVA, IVA, Placebo, Placebo |
| NCT03911713 A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis | Cystic Fibrosis | P2 | Completed | 77 | RCT, Double-blind | VX-561, IVA, Placebo, Placebo |
| NCT00892697 Intrahepatic HCV RNA and Telaprevir Kinetics in Hepatitis C Virus (HCV) | Hepatitis C | P2 | Completed | 15 | Open-label | Telaprevir, Peginterferon alfa-2a, Ribavirin |
| NCT00003847 VX-710, Doxorubicin, and Vincristine for the Treatment of Patients With Recurrent Small Cell Lung Cancer | Lung Cancer | P2 | Terminated | 36 | Open-label | biricodar dicitrate, doxorubicin hydrochloride, vincristine sulfate |
| NCT03625466 A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del | Cystic Fibrosis | P2 | Completed | 51 | RCT, Double-blind | LUM/IVA, LUM/IVA, Placebo |
| NCT03227471 A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis | Cystic Fibrosis | P1P2 | Completed | 225 | RCT, Double-blind | IVA, TEZ/IVA, VX-445, Matched Placebo, TEZ, VX-561, VX-445 |
| NCT02951182 A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis | Cystic Fibrosis | P2 | Completed | 74 | RCT, Double-blind | TEZ, IVA, VX-440, Matched Placebo |
| NCT00621296 Safety and Efficacy of MP-424 to Treat Chronic Hepatitis C | Hepatitis C | P2 | Completed | 15 | Open-label | MP-424 (Telaprevir) |
| NCT00262483 Phase 2 Study of VX-950, Pegasys and Copegus in Hepatitis C | Hepatitis C | P2 | Completed | 12 | Open-label | VX-950, ribavirin, peginterferon alfa-2a |
| NCT01052194 A 12-week Study of 4 Doses of VX-509 in Subjects With Active Rheumatoid Arthritis | Rheumatoid Arthritis | P2 | Completed | 206 | RCT, Double-blind | Placebo, VX-509, VX-509, VX-509, VX-509 |
| NCT01531673 Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation | Cystic Fibrosis | P2 | Completed | 194 | RCT, Double-blind | VX-661, Ivacaftor, Placebo matched to VX-661, Placebo matched to ivacaftor |
| NCT00493441 AVN944 in Combination With Gemcitabine for the Treatment of Pancreatic Cancer | Pancreatic Cancer | P2 | Terminated | 28 | Open-label | AVN944 |
| NCT01842451 A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-135 and Daclatasvir in Subjects With Genotype 1 Chronic Hepatitis C Chronic Hepatitis C | Chronic Hepatitis C, CHC | P2 | Completed | 23 | RCT, Double-blind | VX-135, Daclatasvir |
| NCT02871778 Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia | Primary Ciliary Dyskinesia | P2 | Completed | 123 | RCT, Double-blind | VX-371, Hypertonic Saline, Placebo (0.17% saline), VX-371 + HS, Ivacaftor |
| NCT00205478 Phase 2 Clinical Study in Rheumatoid Arthritis With an Investigational Oral p38 MAP Kinase Inhibitor VX-702 | Rheumatoid Arthritis | P2 | Completed | 300 | RCT, Double-blind | VX-702 |
| NCT03163511 A Safety, Tolerability, and Efficacy Study of VC-02™ Combination Product in Subjects With Type 1 Diabetes Mellitus and Hypoglycemia Unawareness | Type 1 Diabetes Mellitus With Hypoglycemia | P1P2 | Completed | 49 | Open-label | — |
| NCT03912233 A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis | Cystic Fibrosis | P2 | Completed | 87 | RCT, Double-blind | VX-121, TEZ, VX-561, TEZ/IVA, IVA, Placebo |
| NCT00088504 Merimepodib (MMPD) in Triple Combination for the Treatment of Chronic Hepatitis C | Hepatitis C, Hepatitis | P2 | Completed | 315 | RCT, Double-blind | Merimepodib, PEG-Interferon-alpha 2a (Pegasys®), Ribavirin (Copegus®) |
| NCT00911963 Pharmacokinetics of Multiple Ascending Doses of VCH-222 in Subjects With Chronic Hepatitis C Infection | Hepatitis C | P1P2 | Completed | 49 | RCT, Double-blind | VCH-222 or matching placebo, VCH-222 or matching placebo, peginterferon alfa-2a, ribavirin |
| NCT02343445 Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF) | Cystic Fibrosis | P2 | Completed | 142 | RCT, Double-blind | P-1037, Hypertonic Saline, Saline |
| NCT01590459 24-week Study With Open Label Extension of VX-509, an Oral JAK3 Inhibitor, in Subjects Taking Methotrexate | Rheumatoid Arthritis | P2 | Completed | 359 | RCT, Double-blind | VX-509, VX-509 matching placebo |
| NCT04340362 Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis | Glomerulosclerosis, Focal Segmental | P2 | Completed | 16 | Open-label | VX-147 |
| NCT01080222 A Safety and Efficacy Study of the Combination of VX-222 and Telaprevir in Treatment-Naïve Subjects With Genotype 1 Chronic Hepatitis C Virus Infection | Chronic Hepatitis C Virus Infection | P2 | Terminated | 152 | RCT, Open-label | telaprevir, VX-222, ribavirin, peginterferon-alfa-2a, VX-222 |
| NCT03764072 A Dose-ranging Study to Evaluate Efficacy and Safety of VX-150 in Subjects With Acute Pain Following Bunionectomy | Acute Pain | P2 | Completed | 250 | RCT, Double-blind | VX-150, Placebo |
| NCT05643495 A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype | Alpha-1 Antitrypsin Deficiency | P2 | Terminated | 14 | Open-label | VX-864 |
| NCT00500812 A Safety Study for Cethrin (BA-210) in the Treatment of Acute Thoracic and Cervical Spinal Cord Injuries | Spinal Cord Injury | P1P2 | Completed | 48 | Open-label | Cethrin |
| NCT00865904 Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation | Cystic Fibrosis | P2 | Completed | 93 | RCT, Double-blind | VX-809, Placebo |
| NCT00389298 A Proof-of-concept Study of VCH-759 for the Treatment of Hepatitis C-infection. | Hepatitis C, Chronic | P1P2 | Completed | 36 | RCT, Double-blind | VCH-759 (BCH-27759) |
| NCT05034952 A Study Evaluating Efficacy and Safety of VX-548 for Acute Pain After an Abdominoplasty | Acute Pain | P2 | Completed | 303 | RCT, Double-blind | VX-548, HB/APAP, Placebo (matched to VX-548), Placebo (matched to HB/APAP) |
| NCT03085485 The Topic Trial - Study to Determine the Safety and Efficacy of Ivacaftor | Chronic Obstructive Pulmonary Disease, Chronic Bronchitis | P2 | Completed | 40 | RCT, Double-blind | Ivacaftor 150 MG, Placebo |
| NCT03304522 A Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy | Small Fiber Neuropathy | P2 | Completed | 89 | RCT, Double-blind | VX-150, Placebo |
| NCT01561807 A Study to Investigate VX-787 Given to Adult Volunteers Inoculated With Live Influenza Virus | Influenza Virus | P2 | Completed | 140 | RCT, Double-blind | VX-787, Placebo |
| NCT02971839 Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations | Cystic Fibrosis | P2 | Terminated | 11 | RCT, Double-blind | VX-561, Placebo, IVA |
| NCT06926621 A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I | Myotonic Dystrophy Type 1 (DM1) | P2 | By Invite | 44 | Open-label | VX-670 |
| NCT02508207 A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation | Cystic Fibrosis | P2 | Completed | 34 | RCT, Double-blind | Tezacaftor/Ivacaftor, Ivacaftor, Tezacaftor/Ivacaftor matching placebo, Ivacaftor matching placebo |
| NCT01726946 A Phase 2 Study to Evaluate the Safety and Efficacy of VX-135 With Ribavirin in Treatment-Naïve Subjects With Chronic Hepatitis C | Chronic Hepatitis C | P2 | Completed | 10 | RCT, Double-blind | VX-135, ribavirin |
| NCT01590407 First in Human Study of ALS-002200; Single Dose, Food Effect in Healthy Volunteers; Multiple Doses in Chronic Hepatitis C Genotype 1 | Hepatitis C, Chronic | P1 | Completed | 71 | RCT, Double-blind | ALS-002200, Placebo |
| NCT01704521 Viral Kinetics in HCV Clearance in Subjects With Hemophilia | Chronic Hepatitis C, Hemophilia | P1 | Completed | 5 | RCT, Open-label | PegInterferon, Ribavirin, Telaprevir |
| NCT01554085 First in Human Study of ALS-002158; Single Dose, Food Effect in Healthy Volunteers; Multiple Doses in Chronic Hepatitis C Genotype 1 | Hepatitis C, Chronic | P1 | Terminated | 78 | RCT, Double-blind | ALS-002158, Placebo |
| NCT03486236 A Study Evaluating Safety and Pharmacokinetics of VX-440 in Combination With Tezacaftor/Ivacaftor in Healthy Adult Subjects | Cystic Fibrosis | P1 | Completed | 16 | RCT, Double-blind | VX-440, TEZ, IVA, Matched Placebos |
| NCT01275599 Drug-Drug Interaction Study Between Telaprevir and Buprenorphine | Hepatitis C, Opioid-Related Disorders | P1 | Completed | 16 | Open-label | telaprevir, buprenorphine/naloxone |
| NCT05635110 Evaluation of the Effects of Omeprazole and Rifampin on the Pharmacokinetics of VX-548 in Healthy Participants | Pain | P1 | Completed | 31 | Open-label | VX-548, Omeprazole, Rifampin |
| NCT00251199 VX-950 and Peginterferon for Hepatitis C | Hepatitis C | P1 | Completed | 20 | RCT, Double-blind | VX-950, peginterferon |
| NCT06615570 A Phase 1 Dose Escalation Study of VX-973 in Healthy Participants | Pain | P1 | Completed | 30 | RCT, Double-blind | VX-973, Placebo |
| NCT01144936 Study of VX-985 in Subjects With Chronic Hepatitis C | Chronic Hepatitis C | P1 | Completed | 21 | RCT, Double-blind | VX-985 or matching placebo, VX-985 or matching placebo, VX-985 or matching placebo |
| NCT06861413 A Phase 1 Study to Evaluate the Relative Bioavailability of a VX-828 Tablet Formulation | Cystic Fibrosis | P1 | Completed | 68 | RCT, Open-label | VX-828, VX-828 |
| NCT06820307 Evaluation of the Effect of Suzetrigine (SUZ) on the Pharmacokinetics of Oral Contraceptives in Healthy Female Participants | Pain | P1 | Completed | 50 | Open-label | Suzetrigine, DRSP/EE, NGM/EE |
| NCT05518734 A Phase 1 Dose Escalation Study of VX-708 in Healthy Participants | Pain | P1 | Completed | 64 | RCT, Double-blind | VX-708, Itraconazole, Midazolam, Placebo |
| NCT05865171 A Study to Evaluate Pharmacokinetics (PK) and Safety of Inaxaplin in Participants With Renal Impairment | Renal Impairment | P1 | Completed | 16 | RCT, Open-label | IXP |
| NCT01768663 A Phase 1 Study to Examine the Drug-Drug Interaction of Ciprofloxacin, Itraconazole, and Rifampin on the Combination of Lumacaftor With Ivacaftor in Healthy Adult Subjects | Cystic Fibrosis | P1 | Completed | 80 | Open-label | Lumacaftor, Ivacaftor, Ciprofloxacin, Itraconazole, Rifampin |
| NCT05653323 A Phase 1 Dose Escalation Study of VX-993 in Healthy Participants | Pain | P1 | Completed | 70 | RCT, Double-blind | VX-993, Placebo |
| NCT06226454 A Phase 1 Study Evaluating the Safety, Pharmacokinetics, and Pharmacodynamics, of VX-993 | Pain | P1 | Completed | 39 | RCT, Double-blind | VX-993, Placebo |
| NCT05579431 A Phase 1, First-in-human Study of VX-634 | Alpha 1-Antitrypsin Deficiency | P1 | Completed | 127 | RCT, Double-blind | VX-634, Placebo |
| NCT05560464 Evaluation of the Pharmacokinetics and Safety of VX-548 in Participants With Mild or Moderate Hepatic Impairment | Pain | P1 | Completed | 36 | Open-label | VX-548 |
| NCT00623649 Safety,Tolerability and Pharmacokinetics of Multiple Ascending Doses of VCH 916 in Subjects With Chronic Hep C Infection | HCV Infection | P1 | Completed | 42 | RCT, Double-blind | VCH 916, Placebo |
| NCT00591214 Safety and PK Study of MP-424 to Treat Chronic Hepatitis C | Chronic Hepatitis C | P1 | Completed | 10 | Open-label | MP-424 (Telaprevir) |
| NCT05704556 Evaluation of the Pharmacokinetics and Safety of VX-548 in Participants With Renal Impairment | Pain | P1 | Completed | 38 | Open-label | VX-548 |
| NCT01216046 Drug-Drug Interaction Study of VX-770 and VX-809 in Healthy Subjects | Cystic Fibrosis | P1 | Completed | 48 | RCT, Double-blind | VX-809, VX-770, VX-809 placebo, VX-770 placebo |
| NCT00958152 Drug-Drug Interaction Study of VCH-222 and Telaprevir in Healthy Subjects | Hepatitis C | P1 | Completed | 30 | RCT, Double-blind | VCH-222, VCH-222, VCH-222, telaprevir |
| NCT00509210 Study of Telaprevir in Subjects With Hepatic Impairment | Hepatic Insufficiency | P1 | Completed | — | Open-label | telaprevir (VX-950) |
| NCT05541471 A Study to Evaluate the Pharmacokinetic Drug-drug Interactions Between VX-548, Midazolam, and Digoxin | Pain | P1 | Completed | 37 | Open-label | VX-548, Midazolam, Digoxin |
| NCT01886209 Phase 1 PK Interaction Study Between VX-509 and Prednisone or Methylprednisolone in Healthy Male Subjects | Drug Interactions | P1 | Completed | 28 | Open-label | Prednisone, VX-509, Methylprednisolone |
| NCT07074327 Effects of VX-407 on the Pharmacokinetics of Oral Contraceptives in Healthy Participants | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | P1 | Completed | 74 | Open-label | VX-407, LNG/EE, NGM/EE, NET/EE, DRSP/EE |
| NCT06886269 A Study to Evaluate How the Body Processes 14C-VX-993 After a Single Oral Dose | Pain | P1 | Completed | 8 | Open-label | 14C-VX-993 |
| NCT01208285 Study of VX-770 in Subjects With Moderate Hepatic Impairment and in Matched Healthy Subjects | In Development for Cystic Fibrosis | P1 | Completed | 24 | Open-label | VX-770 |
| NCT06392659 A Phase 1, Open-label Study Evaluating the Pharmacokinetics and Drug-drug Interaction of VX-993 in Healthy Adults | Pain | P1 | Completed | 42 | Open-label | VX-993, Gemfibrozil, Itraconazole, Midazolam |
| NCT05867147 An Electrocardiogram Study to Evaluate the Effect of Vanzacaftor on the QT/QTc Interval in Healthy Participants | Cystic Fibrosis | P1 | Completed | 56 | RCT, Double-blind | Vanzacaftor, Vanzacaftor Placebo, Moxifloxacin, Moxifloxacin Placebo |
| NCT00273936 Trial of AVN-944 in Patients With Advanced Hematologic Malignancies | Acute Leukemia, Chronic Leukemia | P1 | Completed | 72 | Open-label | AVN-944 capsules for oral administration |
| NCT05818852 A Study to Evaluate the Effect of VX-548 on the QT/QTc Interval in Healthy Participants | Pain | P1 | Completed | 72 | RCT, Double-blind | VX-548, Moxifloxacin, Moxifloxacin Placebo, VX-548 Placebo |
| NCT06299709 A Study To Evaluate the Relative Bioavailability, Food Effect, and Dose Proportionality of a Granule Formulation of Vanzacaftor/Tezacaftor/Deutivacaftor(VNZ/TEZ/D-IVA) | Cystic Fibrosis | P1 | Completed | 34 | RCT, Open-label | VNZ/TEZ/D-IVA, VNZ/TEZ/D-IVA |
| NCT06834009 A Study of Suzetrigine (SUZ) Spray-dried Dispersion (SSD) in Healthy Adult Panelists | Pain | P1 | Completed | 10 | Open-label | SUZ |
| NCT05727800 A Phase 1, First-in-human Study of VX-668 | Alpha-1 Antitrypsin Deficiency | P1 | Completed | 114 | RCT, Double-blind | VX-668, Placebo |
| NCT06312787 A Study to Evaluate the Relative Bioavailability and Food Effect of a VX-118 Tablet Formulation | Cystic Fibrosis | P1 | Completed | 15 | RCT, Open-label | VX-118 |
| NCT05437120 Pharmacokinetics and Safety Assessment of VX-121/Tezacaftor/Deutivacaftor in Participants With Moderate Hepatic Impairment | Cystic Fibrosis | P1 | Completed | 16 | Open-label | VX-121/TEZ/D-IVA |
| NCT05347394 A Phase 1 Dose Escalation Study of Oral VX-708 in Healthy Participants | Acute Pain | P1 | Completed | 100 | RCT, Double-blind | VX-708, Placebo |
| NCT05866055 A Phase 1 Dose Escalation Study of VX-973 in Healthy Adults | Pain | P1 | Completed | 30 | RCT, Double-blind | VX-973, Placebo, Midazolam |
| NCT05955872 A Study Evaluating the Relative Bioavailability and Food Effect of a Tablet Formulation of VX-147 | Focal Segmental Glomerulosclerosis (FSGS) | P1 | Completed | 21 | RCT, Open-label | VX-147 |
| NCT06420765 A Microneurography Study of NaV1.8 Inhibition in Healthy Adults | Pain | P1 | Completed | 92 | RCT, Double-blind | VX-150, VX-548, VX-993, Placebo |
| NCT06299696 A Study of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) in Healthy Adult Panelists | Cystic Fibrosis | P1 | Completed | 10 | Open-label | VNZ/TEZ/D-IVA |
| NCT06529796 Evaluation of the Pharmacokinetics and Safety of Inaxaplin in Participants With Mild or Moderate Hepatic Impairment | Focal Segmental Glomerulosclerosis (FSGS) | P1 | Completed | 36 | Open-label | IXP |
| NCT00789126 Single and Multiple Dose Escalation Study of VX-509 in Healthy Subjects | Healthy | P1 | Completed | 72 | RCT, Double-blind | VX-509 |
| NCT00973388 VX-950-TiDP24-C136 - A Trial to Evaluate the Effect of Telaprevir (TVR) on the Results of Electrocardiograms (Electric Recording of the Heart) in Healthy Volunteers | Hepatitis C | P1 | Completed | 44 | RCT, Double-blind | Telaprevir; Moxifloxacin; Placebo |
| NCT01018368 Study of VX-770 and Rifampin in Healthy Male Subjects | Cystic Fibrosis | P1 | Completed | 24 | Open-label | VX-770, Rifampin |
| NCT00933283 A Study to Investigate the Potential Interaction Between Telaprevir and Methadone, at Steady-State | Healthy Participants | P1 | Completed | 18 | Open-label | Telaprevir, Methadone |
| NCT07022119 A Phase 1 Study Evaluating Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | P1 | Completed | 32 | RCT, Double-blind | VX-407, Placebo |
| NCT02015507 An Open-Label, Phase 1 Study in Healthy Adult Subjects to Examine the Effects of Multiple-Dose Ciprofloxacin on Ivacaftor and VX-661 in Combination With Ivacaftor | Cystic Fibrosis | P1 | Completed | 34 | Open-label | ivacaftor, VX-661, ciprofloxacin |
| NCT05455502 A Study to Evaluate the Relative Bioavailability and Food Effect of a New Tablet Formulation of VX-548 | Pain | P1 | Completed | 24 | RCT, Open-label | VX-548 |
| NCT06523595 Evaluation of the Pharmacokinetic Drug-drug Interactions Between VX-993 and Metformin in Healthy Adults | Pain | P1 | Completed | 31 | Open-label | VX-993, Metformin |
| NCT06345755 A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | P1 | Completed | 159 | RCT, Double-blind | VX-407, Placebo, Midazolam, VX-407 |
| NCT00630058 A Phase 1 Study of MP-424, Peginterferon Alfa 2b, and Ribavirin in Hepatitis C | Hepatitis C | P1 | Completed | 20 | RCT, Open-label | MP-424(H), PEG-IFN-a-2b, RBV, MP-424 (L), PEG-IFN-a-2b, RBV |
| NCT03029455 A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis | Cystic Fibrosis | P1 | Completed | 163 | RCT, Double-blind | VX-659, Tezacaftor, Ivacaftor, VX-659 Matching Placebo, Triple Combination (TC) Matching Placebos |
| NCT01888393 Study of Lumacaftor in Combination With Ivacaftor in Subjects With Moderate Hepatic Impairment and Healthy Subjects | Hepatic Impairment, Healthy | P1 | Completed | 23 | Open-label | lumacaftor 200 mg q12h + ivacaftor 250 mg q12h |
| NCT01253551 VX-950HEP1001 - Drug-drug Interaction Study Between Telaprevir and Raltegravir | Hepatitis C | P1 | Completed | 21 | RCT, Open-label | Treatment sequence AB, Treatment sequence BA |
| NCT01511432 A Study to Assess the Relative Bioavailability Three New Formulations of Telaprevir in Healthy Subjects | Chronic Hepatitis C | P1 | Completed | 64 | RCT, Open-label | telaprevir formulation A, telaprevir Formulation B, telaprevir Formulation C, telaprevir Formulation D |
| NCT05851157 A Study to Evaluate the Effect of Food on the Pharmacokinetics of VX-548 | Pain | P1 | Completed | 73 | Open-label | VX-548 |
| NCT01899105 A Phase 1 Study to Investigate the Food Effect of Lumacaftor in Combination With Ivacaftor | Cystic Fibrosis | P1 | Completed | 28 | RCT, Open-label | lumacaftor, ivacaftor |
| NCT01910415 Phase 1, QT/QTC Interval Study in Healthy Subjects | Cystic Fibrosis | P1 | Completed | 200 | RCT, Double-blind | Lumacaftor, Lumacaftor Placebo, Ivacaftor, Ivacaftor Placebo, moxifloxacin hydrochloride |
| NCT00966602 Drug-Drug Interaction Study of VX-809 and VX-770 in Healthy Subjects | Cystic Fibrosis | P1 | Completed | 24 | RCT, Double-blind | VX-809, VX-770, VX-809 & VX-770, Placebo |
| NCT06336096 A Study to Evaluate the Relative Bioavailability and Food Effect of a New Tablet Formulation and Strength of Suzetrigine | Pain | P1 | Completed | 48 | RCT, Open-label | Suzetrigine, Suzetrigine |
| NCT05324410 A Phase 1 Dose Escalation Study to Evaluate Safety and Pharmacokinetics (PK) of VX-840 in Healthy Participants | APOL1-mediated Kidney Disease | P1 | Completed | 88 | RCT, Double-blind | VX-840, Placebo |
| NCT07010406 A Study to Evaluate the Relative Bioavailability of Povetacicept Formulations and Bioequivalence of Povetacicept Presentations | Healthy Volunteers | P1 | Completed | 136 | RCT, Open-label | Povetacicept |
| NCT01060566 Study of VX-770 on Midazolam and Rosiglitazone and the Effect of Fluconazole on VX-770 | Cystic Fibrosis | P1 | Completed | 24 | Open-label | VX-770, VX-770 |
| NCT06394167 A Phase 1 Dose Escalation Study of Intravenous VX-993 in Healthy Adults | Pain | P1 | Completed | 88 | RCT, Double-blind | VX-993, Placebo |
| NCT05535959 A Study to Evaluate the Relative Bioavailability of a Fixed-dose Combination Tablet of VX-121/Tezacaftor/Deutivacaftor | Cystic Fibrosis | P1 | Completed | 16 | RCT, Open-label | VX-121/TEZ/D-IVA |
| NCT01038167 A Study to Examine the Effects of Telaprevir on the Pharmacokinetics of Cyclosporine and Tacrolimus in Healthy Adults | Hepatitis C | P1 | Completed | 20 | Open-label | telaprevir, telaprevir, cyclosporine, cyclosporine, tacrolimus, tacrolimus |
| NCT06508762 A Study to Evaluate the Relative Bioavailability and Food Effect of a New Tablet Formulation of VX-993 | Pain | P1 | Completed | 24 | Open-label | VX-993 |
| NCT05418712 A Microneurography (MNG) Study of VX-150 in Healthy Participants | Pain | P1 | Completed | 45 | RCT, Double-blind | Placebo, VX-150 |
| NCT01153542 Study of VX-770 on Desipramine | In Development for Cystic Fibrosis | P1 | Completed | 24 | Open-label | VX-770, VX-770 |
| NCT07349394 Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants | Cystic Fibrosis | P1 | Completed | 18 | Open-label | VNZ/TEZ/D-IVA, Rosuvastatin |
| NCT03304028 MUCOviscidose EXacerbation Outils Connectés Education Thérapeutique | Cystic Fibrosis | N/A | Terminated | 36 | Open-label | — |
| NCT03659214 Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults | Cystic Fibrosis | Completed | 19 | — | — | |
| NCT04138589 Effect of Lumacaftor/Ivacaftor in Children With Cystic Fibrosis Homozygote for F508del on Small Airway Function | Cystic Fibrosis in Children | Completed | 30 | — | — | |
| NCT03783286 Ivacaftor Treatment in 4 Month to 2 Year Old CF Subjects | Cystic Fibrosis | Completed | 15 | — | Ivacaftor | |
| NCT03859531 Gastrointestinal Study at Orkambi Therapy in CF Patients | Cystic Fibrosis | Unknown | 20 | — | — | |
| NCT00916474 Virology Follow up Study in Subjects Previously Treated With Telaprevir | Hepatitis C | Completed | 408 | — | telaprevir | |
| NCT04702360 ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation | Cystic Fibrosis | APPROVED_FOR_MARKETING | — | — | ELX/TEZ/IVA, IVA | |
| NCT01863238 An Ocular Safety Study of Ivacaftor-Treated Pediatric Patients 11 Years of Age or Younger With Cystic Fibrosis | Cystic Fibrosis | Completed | 95 | — | Ivacaftor Exposed | |
| NCT03278314 Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis | Cystic Fibrosis | APPROVED_FOR_MARKETING | — | — | tezacaftor/ivacaftor | |
| NCT03338595 Longitudinal Assessment of Exercise Capacity and Vascular Function in Patients With CF | Cystic Fibrosis | Completed | 20 | — | — | |
| NCT01381289 VX-770 Expanded Access Program | Cystic Fibrosis | APPROVED_FOR_MARKETING | — | — | VX-770 | |
| NCT03227094 Simplification of CF-related Diabetes Screening at Home | Cystic Fibrosis, Cystic Fibrosis-related Diabetes | N/A | Completed | 37 | RCT, Open-label | — |
| NCT06506773 Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF) | Cystic Fibrosis | Completed | 79 | — | — | |
| NCT04058210 VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes) | Cystic Fibrosis | APPROVED_FOR_MARKETING | — | — | ELX/TEZ/IVA, IVA | |
| NCT04618185 Gut Imaging for Function & Transit in CF - GIFT-CF 3 | Cystic Fibrosis | Completed | 24 | — | — | |
| NCT02141464 Energy Balance and Weight Gain With Ivacaftor Treatment | Cystic Fibrosis | Completed | 24 | — | — | |
| NCT02445053 Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) | Cystic Fibrosis | Completed | 75 | — | ivacaftor | |
| NCT04254705 Organoid Study R334W | Cystic Fibrosis | N/A | Withdrawn | — | Open-label | — |
| NCT04923464 A Decentralized Study to Evaluate Physical Activity and Cough Frequency Using Wearable Technology in Cystic Fibrosis | Cystic Fibrosis | Completed | 51 | — | — | |
| NCT06147778 Performances and Safety of MuCopilot, a Digital Tool for the Unsupervised Objective Assessment of Cystic Fibrosis | Cystic Fibrosis | N/A | Completed | 70 | Open-label | — |
| NCT00923728 A Phase I Trial of AVN944, an IMPDH Inhibitor, in Adults With Advanced Stage Solid Tumors | Refractory Solid Tumors | N/A | Withdrawn | — | Open-label | AVN944 |
| NCT02722057 A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients | Cystic Fibrosis | Completed | 368 | — | — | |
| NCT03795363 Orkambi Treatment in 2 to 5 Year Old Children With CF | Cystic Fibrosis | Completed | 28 | — | Orkambi |