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Clinical trial

An Operationally Seamless Phase 1/2/3 Study Consisting of a Safety and Dose-finding Phase 1/2 and Randomized, Open-label, Active-controlled Phase 3 to Evaluate UX701 AAV Gene Therapy in Adults With Wilson Disease

NCT04884815 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.

NCT NCT04884815
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Registry record

As filed in ClinicalTrials.gov.

Title
An Operationally Seamless Phase 1/2/3 Study Consisting of a Safety and Dose-finding Phase 1/2 and Randomized, Open-label, Active-controlled Phase 3 to Evaluate UX701 AAV Gene Therapy in Adults With Wilson Disease
Lead sponsor
Ultragenyx Pharmaceutical Inc
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
September 27, 2021
Completion date
March 31, 2034
Enrollment
82 (estimated)
Sites
16

Conditions

As the registry lists them; a condition with its own page is linked.

wilson disease