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Clinical trial

A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)

NCT04259281 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.

NCT NCT04259281
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)
Lead sponsor
Ultragenyx Pharmaceutical Inc
Phase
Phase 1/2
Overall status
Completed
Start date
February 24, 2020
Completion date
January 8, 2025
Enrollment
74 (actual)
Sites
25

Conditions

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

angelman syndrome