Phase 2
N=11
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
Ornithine Transcarbamylase (OTC) Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT02991144 ↗Enrolled (actual)
11
Serious AEs
9.1%
Results posted
Dec 2022
Primary outcome: Primary: Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation — 1; 1; 0; 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- scAAV8OTC (Genetic); Reactive Corticosteroid Taper Regimen (Drug); Prophylactic Corticosteroid Taper Regimen (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Ultragenyx Pharmaceutical Inc
- Primary completion
- Dec 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation |
1; 1; 0; 1; 3; 3 | — |
| SECONDARY Change From Baseline Over Time in Rate of Ureagenesis |
41.139; 46.857; -29.017; 14.586; 2.363; 20.897 | — |
| SECONDARY Change From Baseline Over Time in Area Under the Curve From Time Zero to 24 Hours (AUC0-24) of Plasma Ammonia |
97.69; -1334.27; -3790.83; -1095.20; -264.07; -1384.95 | — |
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.
Eligibility Criteria
Key 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 2.0 mg/dL.
- Participation in another investigational medicine study (including another gene transfer trial) within 3 months of Screening
- Pregnant or nursing
Note additional inclusion/exclusion criteria may apply, per protocol.
Data sourced from ClinicalTrials.gov (NCT02991144). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.