Sarepta Therapeutics Inc
SRPTHealthcare|Biotechnology|USA
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Clinical Trials (55)
| Trial | Conditions | Phase | Status | Enrollment | Design | Drug |
|---|---|---|---|---|---|---|
| NCT03992430 A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) | Muscular Dystrophy, Duchenne | P3 | Active | 160 | RCT, Double-blind | Eteplirsen |
| NCT05881408 A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | P3 | Active | 148 | RCT, Double-blind | — |
| NCT06246513 A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 | Limb-girdle Muscular Dystrophy | P3 | Active | 17 | Open-label | SRP-9003, Glucocorticoid |
| NCT06128564 A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | P2 | Active | 13 | Open-label | — |
| NCT04626674 A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) | Muscular Dystrophy, Duchenne | P1 | Recruiting | 83 | Open-label | — |
| NCT05876780 A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency) | Limb Girdle Muscular Dystrophy | P1 | Active | 6 | Open-label | — |
| NCT06581887 Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies | DMD, BMD | Recruiting | 100 | — | — | |
| NCT04475926 A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice | Limb-girdle Muscular Dystrophy | Active | 205 | — | — | |
| NCT04708314 An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | P4 | Terminated | 2 | Open-label | Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53] |
| NCT03532542 An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | P3 | Terminated | 171 | Open-label | Casimersen, Golodirsen |
| NCT06952686 A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants | Muscular Dystrophies, Limb-Girdle | P3 | Withdrawn | — | Open-label | SRP-9005, Corticosteroid |
| NCT05096221 A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | P3 | Completed | 126 | RCT, Double-blind | — |
| NCT02500381 Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | P3 | Completed | 228 | RCT, Double-blind | SRP-4045, SRP-4053, Placebo |
| NCT02255552 Study of Eteplirsen in DMD Patients | Duchenne Muscular Dystrophy (DMD) | P3 | Completed | 109 | Open-label | eteplirsen |
| NCT05967351 A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study | Duchenne Muscular Dystrophy | P3 | By Invite | 400 | Open-label | — |
| NCT03769116 A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD) | Muscular Dystrophy, Duchenne | P1P2 | Completed | 41 | RCT, Double-blind | — |
| NCT00844597 Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients | Duchenne Muscular Dystrophy | P1P2 | Completed | 19 | Open-label | AVI-4658 for Injection |
| NCT01976091 A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D) | Limb-Girdle Muscular Dystrophy, Type 2D | P1P2 | Completed | 6 | Open-label | — |
| NCT03375164 A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | P1P2 | Completed | 4 | Open-label | — |
| NCT01396239 Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients | Duchenne Muscular Dystrophy | P2 | Completed | 12 | RCT, Double-blind | AVI-4658 (Eteplirsen) |
| NCT00229749 Study of AVI-4065 in Healthy Volunteers and Chronic Active HCV Patients | Hepatitis C | P1P2 | Terminated | 80 | Open-label | AVI-4065 Injection |
| NCT04179409 A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. | Duchenne Muscular Dystrophy | P2 | Completed | 3 | Open-label | Amondys 45, Exondys 51, Vyondys 53 |
| NCT03985878 A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995) | Duchenne Muscular Dystrophy | P2 | Terminated | 15 | Open-label | Eteplirsen |
| NCT00159250 Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | P1P2 | Completed | 7 | Single-blind | AVI-4658 (PMO) |
| NCT00248066 Safety and Efficacy of RESTEN-MP When Used in Conjunction With a Bare Metal Stent in Coronary Arteries | Coronary Artery Disease, Coronary Restenosis | P2 | Completed | 50 | Open-label | RESTEN-MP |
| NCT03675126 An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen) | Muscular Dystrophy, Duchenne | P1P2 | Terminated | 15 | Open-label | SRP-5051 |
| NCT02286947 Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | P2 | Completed | 24 | Open-label | Eteplirsen |
| NCT03218995 Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy | P2 | Completed | 15 | Open-label | Eteplirsen |
| NCT02310906 Phase I/II Study of SRP-4053 in DMD Patients | Duchenne Muscular Dystrophy | P1P2 | Completed | 39 | RCT, Double-blind | Placebo, SRP-4053 |
| NCT03652259 Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2E | P1P2 | Terminated | 6 | Open-label | — |
| NCT02420379 Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD) | P2 | Completed | 33 | Open-label | eteplirsen |
| NCT04004065 Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | Duchenne Muscular Dystrophy | P2 | Terminated | 62 | RCT, Open-label | Vesleteplirsen |
| NCT01540409 Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD) | P2 | Completed | 12 | Open-label | AVI-4658 (Eteplirsen) |
| NCT00451256 Safety and Efficacy Study of AVI-5126 When Used on Vein Grafts Before Use in Heart by-Pass Graft Surgery (CABG) | Cardiovascular Disease, Coronary Artery Bypass | P2 | Terminated | 600 | RCT, Double-blind | AVI-5126 |
| NCT02530905 Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients | Duchenne Muscular Dystrophy | P1 | Completed | 12 | RCT, Double-blind | SRP-4045, Placebo |
| NCT01353027 Safety Study of Single Administration Post-Exposure Prophylaxis Treatment for Ebola Virus | Ebola Hemorrhagic Fever | P1 | Completed | 30 | RCT, Double-blind | Placebo, AVI-6002 |
| NCT00343148 Pharmacokinetic Study of a Single Dose of AVI-4126 (RESTEN-NG®) in Cerebral Spinal Fluid | Neoplasms | P1 | Completed | 6 | Open-label | AVI-4126 Injection (RESTEN-NG®) |
| NCT01353040 Safety Study of Single Administration Post-exposure Prophylaxis Treatment for Marburg Virus | Marburg Hemorrhagic Fever | P1 | Completed | 30 | RCT, Double-blind | AVI-6003, Placebo |
| NCT01375985 Safety Study of Single Administration Intravenous Treatment for Influenza | Influenza | P1 | Terminated | 8 | RCT, Double-blind | AVI-7100, Placebo |
| NCT06747273 Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States | Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3 | P1 | Terminated | 4 | Open-label | SRP-9004 |
| NCT03375255 A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD) | Muscular Dystrophy, Duchenne | P1 | Completed | 15 | Open-label | SRP-5051 |
| NCT01593072 A Study to Assess the Safety, Tolerability and Pharmacokinetics of AVI-7537 in Healthy Adult Volunteers | Ebola Hemorrhagic Fever | P1 | Withdrawn | — | RCT, Double-blind | AVI-7537 |
| NCT00244647 A Phase 1b Study Evaluating RESTEN-MP in Subjects With Focal de Novo Stenosis | Coronary Artery Disease, Coronary Stent Restenosis | P1 | Terminated | 40 | RCT, Single-blind | RESTEN-MP |
| NCT06241950 A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74) | Duchenne Muscular Dystrophy | P1 | Terminated | 5 | Open-label | imlifidase |
| NCT00387283 Pharmacokinetic Study in Cerebral Spinal Fluid After a Single Dose of AVI-4020 | West Nile Virus | P1 | Completed | 14 | Open-label | AVI-4020 Injection |
| NCT05906251 A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related) | Limb Girdle Muscular Dystrophy | P1 | Terminated | 2 | Open-label | — |
| NCT06597656 A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74 | Duchenne Muscular Dystrophy | P1 | Terminated | 3 | Open-label | — |
| NCT00381433 Pharmacokinetic Study of a Single Dose of AVI-4065 in Cerebral Spinal Fluid | Encephalitis | P1 | Completed | 12 | Open-label | AVI-4065 Injection |
| NCT02710500 rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies | Dysferlinopathy | P1 | Completed | 2 | Double-blind | rAAVrh74.MHCK7.DYSF.DV |
| NCT01566877 A Study to Assess the Safety, Tolerability and Pharmacokinetics of AVI-7288 in Healthy Adult Volunteers | Marburg Hemorrhagic Fever | P1 | Completed | 40 | RCT, Double-blind | AVI-7288 |
| NCT00091845 An Exploratory Study of AVI-4020 in Patients With Possible Acute Neuroinvasive West Nile Virus (WNV) Disease | West Nile Fever | P1 | Terminated | 50 | Open-label | AVI-4020 Injection |
| NCT03655223 Early Check: Expanded Screening in Newborns | Spinal Muscular Atrophy, Fragile X Syndrome | By Invite | 30,000 | — | — | |
| NCT06270719 An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | By Invite | 500 | — | Standard of Care | |
| NCT06606340 A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice | Duchenne Muscular Dystrophy | By Invite | 300 | — | Eteplirsen, Golodirsen, Casimersen | |
| NCT03680365 Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families | Duchenne Muscular Dystrophy, Burden, Dependency | Completed | 60 | — | — |