Status:
ACTIVE_NOT_RECRUITING
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Conditions:
OTC Deficiency
Eligibility:
All Genders
12+ years
Phase:
PHASE3
Brief Summary
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.
Detailed Description
This study is a Phase 3, randomized, double-blind, placebo-controlled study of DTX301 in patients with late-onset OTC deficiency 12 years of age and older. Participants will be randomized 1:1 to DTX3...
Eligibility Criteria
Inclusion
- Key
- Confirmed clinical diagnosis of late-onset OTC deficiency with historical documentation by enzymatic (ie, liver biopsy), biochemical (ie, hyperammonemia in the presence of elevated plasma glutamine, low citrulline, and elevated spot urine orotic acid), or molecular testing (ie, OTC analysis)
- Free from symptomatic hyperammonemia and has not required emergent active intervention for hyperammonemia within 4 weeks before screening/baseline
- If on ongoing daily ammonia scavenger therapy, must be at stable daily dose(s) for ≥ 4 weeks prior to screening
- If on a protein-restricted diet, must be on a stable total daily protein intake that does not vary more than 20% for ≥ 4 weeks prior to screening
- From the time written informed consent through Visit 28, females of childbearing potential and fertile males must consent to use highly effective contraception. If female, agree not to become pregnant. If male, agree not father a child or donate sperm
- Key
Exclusion
- Significant hepatic inflammation or cirrhosis
- Estimated glomerular filtration rate \< 60 mL/min/1.73 m2 at screening by the 2021 CKD-EPI creatinine-based formula (Inker et al., 2021) for patients ≥ 18 years of age or the Schwartz bedside formula (Schwartz and Work, 2009) for patients \< 18 years of age
- Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection, documented by current use of antiviral therapy for HBV or HCV or by hepatitis B surface antigen (HBsAg) or HCV RNA positivity
- Active infection (viral or bacterial)
- Detectable pre-existing antibodies to the AAV8 capsid
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results
- Participation (current or previous) in another gene transfer study
- Note: Additional inclusion/exclusion criteria may apply, per protocol
Key Trial Info
Start Date :
October 18 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 1 2031
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT05345171
Start Date
October 18 2022
End Date
March 1 2031
Last Update
December 15 2025
Active Locations (16)
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1
University of California
Los Angeles, California, United States, 90095
2
University of Colorado
Aurora, Colorado, United States, 80045
3
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
4
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44106