Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
CAPtivate
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
3 other identifiers
observational
11
5 countries
9
Brief Summary
Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Aug 2018
Longer than P75 for all trials
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 15, 2018
CompletedFirst Posted
Study publicly available on registry
August 17, 2018
CompletedStudy Start
First participant enrolled
August 30, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2029
February 19, 2026
February 1, 2026
11.3 years
August 15, 2018
February 17, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Participants with Adverse Events and Serious Adverse Events
Up to 416 weeks
Secondary Outcomes (2)
Change from Baseline Over Time in the Ureagenesis Rate
Baseline (average of Screening and Day 1) up to 416 weeks following DTX301 administration
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
Interventions
No Intervention
Eligibility Criteria
Subjects 18 years of age or older with OTC previously enrolled in Study 301OTC01
You may qualify if:
- Completed the Week 52 visit in Study 301OTC01.
- Willing and able to provide written informed consent.
- Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.
You may not qualify if:
- 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.
- Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (9)
The Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Icahn School of Medicine
New York, New York, 10029, United States
University Hospital Cleveland Medical Center/Case Western Reserve University
Cleveland, Ohio, 44106, United States
M.A.G.I.C. Clinic
Calgary, Alberta, T2E 7Z4, Canada
Hopital Femme Mere Enfant
Bron, Rhone, 69677, France
Hospital Clinico Universitario de Santiago
Santiago de Compostela, Coruna, 15706, Spain
Hospital Universitario de Cruces. Servicio de Pediatria
Barakaldo, Vizcaya, 48903, Spain
Queen Elizabeth Hospital, Department of Endocrinology
Birmingham, B15 2TH, United Kingdom
Related Links
Biospecimen
Blood
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Ultragenyx Pharmaceuticals Inc
Study Design
- Study Type
- observational
- Observational Model
- OTHER
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 15, 2018
First Posted
August 17, 2018
Study Start
August 30, 2018
Primary Completion (Estimated)
December 1, 2029
Study Completion (Estimated)
December 1, 2029
Last Updated
February 19, 2026
Record last verified: 2026-02