171. ウィルソン病 Wilson disease Clinical trials / Disease details

臨床試験数 : 79 / 薬物数 : 77 - (DrugBank : 17) / 標的遺伝子数 : 6 - 標的パスウェイ数 : 30

1 trial found

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	EUCTR2020-005266-34-ES (EUCTR)	11/02/2022	06/09/2021	A study to understand the safety and the effects of a virus that transfers a modified protein responsible for copper metabolism (copper-transporting P-type adenosine triphosphatase, ATP7B) in adults with Wilson disease.	A Randomized, Double-blind, Placebo-controlled, Multicenter, Seamless, Adaptive, Safety, Dose-finding, and Phase 3 Clinical Study of UX701 AAV-mediated Gene Transfer for the Treatment of Wilson Disease - Cyprus2+	Wilson disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: UX701 (5.0 × 10^12 genome copies/kg) INN or Proposed INN: UX701 Other descriptive name: Adeno-associated viral vector serotype 9 encoding human ATP7B Product Name: UX701 (1.0 × 10^13 genome copies/kg) INN or Proposed INN: UX701 Other descriptive name: Adeno-associated viral vector serotype 9 encoding human ATP7B Product Name: UX701 (2.0 × 10^13 genome copies/kg) INN or Proposed INN: UX701 Other descriptive name: Adeno-associated viral vector serotype 9 encoding human ATP7B	Ultragenyx Pharmaceutical Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	94	Phase 1;Phase 2;Phase 3	United States;Portugal;Spain;Austria;Italy;United Kingdom;France;Canada;Argentina;Brazil;Poland;Germany;Japan