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
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agemin
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PhaseCountries
1NCT05243017
(ClinicalTrials.gov)
October 7, 20211/11/2021Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's DiseaseA 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 DiseaseHuntington DiseaseGenetic: intra-striatal rAAV5-miHTTUniQure Biopharma B.V.NULLRecruiting25 Years65 YearsAll15Phase 1/Phase 2Germany;Poland;United Kingdom
2EUCTR2020-001461-36-DE
(EUCTR)
13/09/202130/04/2021Safety and Efficacy of AMT-130 in European Adults with Early Manifest Huntington DiseaseA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
15Phase 1;Phase 2Poland;Germany;United Kingdom
3EUCTR2020-001461-36-PL
(EUCTR)
30/06/202125/03/2021Safety and Efficacy of AMT-130 in European Adults with Early Manifest Huntington DiseaseA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
15Phase 1;Phase 2Poland;United Kingdom
4NCT04120493
(ClinicalTrials.gov)
September 6, 201930/9/2019Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's DiseaseA 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 DiseaseHuntington's DiseaseGenetic: intra-striatal rAAV5-miHTT;Other: Imitation (sham) surgeryUniQure Biopharma B.V.NULLRecruiting25 Years65 YearsAll44Phase 1/Phase 2United States