Trials / Active Not Recruiting
Active Not RecruitingNCT03636438
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
A Long-Term Follow-up Study to Evaluate 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
- Status
- Active Not Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 11 (actual)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- —
Summary
Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.
Detailed description
Study 301OTC02 is 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 OTC in adults with late-onset OTC deficiency. Only subjects who complete Study 301OTC01 (NCT02991144) are eligible to participate in Study 301OTC02.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| OTHER | No Intervention | No Intervention |
Timeline
- Start date
- 2018-08-30
- Primary completion
- 2029-12-01
- Completion
- 2029-12-01
- First posted
- 2018-08-17
- Last updated
- 2026-02-19
Locations
9 sites across 5 countries: United States, Canada, France, Spain, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03636438. Inclusion in this directory is not an endorsement.