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 study to understand the safety and the effects of a virus that transfers a modified protein respon ...
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+ A Randomized, Double-blind, Placebo-controlled, Multicenter, Seamless, Adaptive, Safety, Dose-findin ...
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] Wilson disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;S ...
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 ATP7BProduct Name: UX701 (5.0 × 10^12 genome copies/kg) INN or Proposed INN: UX701 Other descriptive name: ...
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 United States;Portugal;Spain;Austria;Italy;United Kingdom;France;Canada;Argentina;Brazil;Poland;Germ ...