Intra-striatal 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2

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.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		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
2NCT04120493
(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