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


Lead Sponsor: Ultragenyx Pharmaceutical Inc

Source Ultragenyx Pharmaceutical Inc
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.

Overall Status Enrolling by invitation
Start Date 2018-08-30
Completion Date 2025-12-01
Primary Completion Date 2025-12-01
Study Type Observational
Primary Outcome
Measure Time Frame
Number of Participants with Adverse Events and Serious Adverse Events Up to 260 weeks following DTX301 administration
Secondary Outcome
Measure Time Frame
Change from Baseline Over Time in the Ureagenesis Rate Baseline (average of Screening and Day 1) up to 260 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 260 weeks following DTX301 administration
Enrollment 18

Intervention Type: Other

Intervention Name: No Intervention

Description: No Intervention


Sampling Method:

Non-Probability Sample


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.



Minimum Age:

18 Years

Maximum Age:


Overall Official
Last Name Role Affiliation
Medical Director Study Director Ultragenyx Pharmaceuticals Inc
Boston Children's Hospital | Boston, Massachusetts, 02115, United States
Icahn School of Medicine | New York, New York, 10029, United States
Alberta Children's Hospital | Calgary, Alberta, T3B 6A8, 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
Location Countries




United Kingdom

United States

Verification Date


Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Acronym CAPtivate
Study Design Info

Observational Model: Other

Time Perspective: Prospective

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