Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarb… (NCT02991144) | Clinical Trial Compass
CompletedPhase 1/2
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
United States, Canada16 participantsStarted 2017-07-31
Plain-language summary
This is a Phase 1/2, open-label, single arm, multicenter, safety and dose finding study of DTX301 in adults with late-onset OTC deficiency. The primary objective of the study is to determine the safety of single intravenous (IV) doses of DTX301.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Males and females ≥18 years of age with documented diagnosis of late onset (defined as first manifestation of signs and symptoms at ≥1 month of age) OTC deficiency, confirmed via enzymatic, biochemical, or molecular testing
✓. Documented history of ≥1 symptomatic hyperammonemia event with ammonia ≥100 µmol/L.
✓. Subject's OTC deficiency is stable as evidenced by either a) no clinical symptoms of hyperammonemia OR b) an ammonia level \<100 µmol/L within the 4 week period preceding the Screening visit.
✓. On ongoing daily stable dose of ammonia scavenger therapy for ≥4 weeks.
✓. Males and all females of childbearing potential must be willing to use effective contraception at the time of administration of gene transfer and for the 52 weeks following administration of DTX301
Exclusion criteria
✕. At Screening or Baseline (Day 0), plasma ammonia level ≥ 100 μmol/L for patients who historically maintain normal ammonia levels; OR plasma ammonia level ≥ 200 μmol/L for patients who historically are not able to fully control ammonia levels with baseline management; OR signs and symptoms of hyperammonemia.
✕. Liver transplant, including hepatocyte cell therapy/transplant.
✕. History of liver disease
✕. Significant hepatic inflammation or cirrhosis
✕. Serum creatinine \>2.0 mg/dL.
✕
What they're measuring
1
Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation
Timeframe: AEs Prior to Dosing: From signing the informed consent form (ICF) to first dose of study drug. TEAEs: From first dose of study drug up to End of Study (Week 52).