RAAV5-miHTT ( DrugBank: rAAV5-miHTT )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 8 | ハンチントン病 | 4 |
8. ハンチントン病
臨床試験数 : 242 / 薬物数 : 205 - (DrugBank : 62) / 標的遺伝子数 : 85 - 標的パスウェイ数 : 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 | EUCTR2020-001461-36-DE (EUCTR) | 13/09/2021 | 30/04/2021 | Safety and Efficacy of AMT-130 in European Adults with Early Manifest Huntington 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 Disease - Safety and Efficacy of AMT-130 in European Adults with Early Manifest Huntington Disease | Huntington Disease MedDRA version: 20.0;Level: PT;Classification code 10070668;Term: Huntington's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: rAAV5-miHTT Product Code: AMT-130 | uniQure biopharma B.V. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 15 | Phase 1;Phase 2 | Poland;Germany;United Kingdom | ||
| 3 | EUCTR2020-001461-36-PL (EUCTR) | 30/06/2021 | 25/03/2021 | Safety and Efficacy of AMT-130 in European Adults with Early Manifest Huntington 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 Disease - Safety and Efficacy of AMT-130 in European Adults with Early Manifest Huntington Disease | Huntington Disease MedDRA version: 20.0;Level: PT;Classification code 10070668;Term: Huntington's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: rAAV5-miHTT Product Code: AMT-130 Other descriptive name: ADENO-ASSOCIATED VIRUS SEROTYPE 5 ENCODING A MICRORNA TARGETED TO HUMAN HUNTINGTIN GENE Product Name: rAAV5-miHTT Product Code: AMT-130 Other descriptive name: ADENO-ASSOCIATED VIRUS SEROTYPE 5 ENCODING A MICRORNA TARGETED TO HUMAN HUNTINGTIN GENE | uniQure biopharma B.V. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 15 | Phase 1;Phase 2 | Poland;United Kingdom | ||
| 4 | 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 |