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Avalotcagene ontaparvovec

DRUG2 trials

Sponsors

Ultragenyx Pharmaceutical Inc.

Conditions

Late-onset Ornithine transcarbamylase (OTC) deficiencyOrnithine transcarbamylase deficiency

Phase 1

A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency
Active, not recruitingCTIS2022-501146-30-00
Ultragenyx Pharmaceutical Inc.Ornithine transcarbamylase deficiency
Start: 2018-09-17Target: 4Updated: 2025-11-18

Phase 3

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus Serotype 8 (AAV8)-mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late-onset OTC Deficiency
Active, not recruitingCTIS2024-514337-38-00
Ultragenyx Pharmaceutical Inc.Late-onset Ornithine transcarbamylase (OTC) deficiency
Start: 2023-01-24Target: 32Updated: 2025-11-25