Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

NCT05345171 · clinicaltrials.gov ↗

PHASE3

Phase

ACTIVE_NOT_RECRUITING

Status

32

Enrollment

INDUSTRY

Sponsor class

Conditions

OTC Deficiency

Interventions

GENETIC: DTX301
OTHER: Placebo
DRUG: Oral Corticosteroids
DRUG: Placebo for oral corticosteroids
DRUG: Sodium Acetate

Sponsor

Ultragenyx Pharmaceutical Inc