UX701 (5.0 × 10^12 genome copies/kg) ( DrugBank: - )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 171 | Wilson disease | 1 |
171. Wilson disease
Clinical trials : 68 / Drugs : 79 - (DrugBank : 17) / Drug target genes : 3 - Drug target pathways : 28
| 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 |