Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency (CAPtivate)

Ultragenyx

Status: Active, not recruiting

Conditions: Ornithine Transcarbamylase (OTC) Deficiency

Treatments: Other: No Intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT03636438

2018-000156-18 (EudraCT Number)

2022-501146-30-00 (EU Trial (CTIS) Number)

301OTC02

Details and patient eligibility

About

Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.

Full 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.

Enrollment: 11 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Completed the Week 52 visit in Study 301OTC01.
2. Willing and able to provide written informed consent.
3. Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.

Exclusion criteria

1. Planned or current participation in another interventional clinical study that may confound the efficacy or safety evaluation of DTX301 during the duration of this study.
2. Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study.