Intra-striatal rAAV5-miHTT ( DrugBank: rAAV5-miHTT )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 8 | Huntington disease | 2 |
8. Huntington disease
Clinical trials : 242 / Drugs : 205 - (DrugBank : 62) / Drug target genes : 85 - Drug target pathways : 159
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05243017 (ClinicalTrials.gov) | October 7, 2021 | 1/11/2021 | Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease | A Phase Ib/II Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Ascending Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT 130) in Early Manifest Huntington's Disease | Huntington Disease | Genetic: intra-striatal rAAV5-miHTT | UniQure Biopharma B.V. | NULL | Recruiting | 25 Years | 65 Years | All | 15 | Phase 1/Phase 2 | Germany;Poland;United Kingdom |
| 2 | NCT04120493 (ClinicalTrials.gov) | September 6, 2019 | 30/9/2019 | Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease | A Phase I/II, Randomized, Double-Blind, Sham Control Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Ascending Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT-130) in Early Manifest Huntington's Disease | Huntington's Disease | Genetic: intra-striatal rAAV5-miHTT;Other: Imitation (sham) surgery | UniQure Biopharma B.V. | NULL | Recruiting | 25 Years | 65 Years | All | 44 | Phase 1/Phase 2 | United States |