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

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03636438

Acronym

CAPtivate

Enrollment

Registered

2018-08-17

Start date

2018-08-30

Completion date

2029-12-01

Last updated

2026-06-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Ornithine Transcarbamylase (OTC) Deficiency

Keywords

gene therapy, OTC Deficiency, Urea Cycle Disorder

Brief 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.

Interventions

OTHERNo Intervention

No Intervention

Study design

Observational model

OTHER

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

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.

Design outcomes

Primary

Measure	Time frame
Number of Participants with Adverse Events and Serious Adverse Events	Up to 416 weeks

Secondary

Measure	Time frame	Description
Change from Baseline Over Time in the Ureagenesis Rate	Baseline (average of Screening and Day 1) up to 416 weeks following DTX301 administration	Sodium acetate is used as a tracer to measure the rate of ureagenesis
Change from Baseline Over Time in 24-Hour Area Under the Curve for Plasma Ammonia	Baseline (Day 0 of Study 301OTC01) up to 208 weeks following DTX301 administration	—

Countries

Canada, France, Spain, United Kingdom, United States

Contacts

STUDY_DIRECTORMedical Director

Ultragenyx Pharmaceuticals Inc

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jun 2, 2026