uniQure NV
QUREHealthcare|Biotechnology|Netherlands
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Tangible Book
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Clinical Trials (11)
| Trial | Conditions | Phase | Status | Enrollment | Design | Drug |
|---|---|---|---|---|---|---|
| NCT04120493 Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease | Huntington's Disease | P1P2 | Active | 43 | Open-label | — |
| NCT06063850 AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy | Mesial Temporal Lobe Epilepsy | P1P2 | Recruiting | 12 | Open-label | — |
| NCT06100276 Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) | Amyotrophic Lateral Sclerosis | P1P2 | Active | 20 | Open-label | AMT-162 |
| NCT06270316 Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease | Fabry Disease | P1P2 | Recruiting | 12 | Open-label | AMT-191 |
| NCT05243017 Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease | Huntington Disease | P1P2 | Active | 14 | RCT, Open-label | — |
| NCT03300453 Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome | Sanfilippo Syndrome B | P1P2 | Completed | 4 | Open-label | rAAV2/5-hNAGLU |
| NCT02904772 Alipogene Tiparvovec for the Treatment of LPLD Patients | LPL Deficiency | P2 | Withdrawn | — | RCT, Open-label | alipogene tiparvovec, Prednisolone, Cyclosporins, Mycophenolate mofetil |
| NCT02082860 Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria | Acute Intermittent Porphyria | P1 | Completed | 8 | Open-label | — |
| NCT02076763 Observational Study of Acute Intermittent Porphyria Patients | Acute Intermittent Porphyria | Completed | 9 | — | — | |
| NCT04723680 An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families | Hemophilia | Completed | 69 | — | — | |
| NCT03293810 Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients | Lipoprotein Lipase Deficiency, Familial Hyperlipoproteinemia Type 1 | Completed | 16 | — | — |