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

Ornithine Transcarbamylase (OTC) Deficiency Clinical Trial

— CAPtivate  

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03636438
• Other study ID #: 301OTC02
• Secondary ID: 2018-000156-1820
• Status: Active, not recruiting
• Start date: August 30, 2018
• Est. completion date: December 2029

Study information

• Verified date: April 2024
• Source: Ultragenyx Pharmaceutical Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 11
• Est. completion date: December 2029
• Est. primary completion date: December 2029
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Ornithine Transcarbamylase (OTC) Deficiency

Intervention

Other:
• No Intervention
No Intervention

Locations

Country	Name	City	State
Canada	Alberta Children's Hospital	Calgary	Alberta
France	Hopital Femme Mere Enfant	Bron	Rhone
Spain	Hospital Universitario de Cruces. Servicio de Pediatria	Barakaldo	Vizcaya
Spain	Hospital Clinico Universitario de Santiago	Santiago De Compostela	Coruna
United Kingdom	Queen Elizabeth Hospital, Department of Endocrinology	Birmingham
United States	The Children's Hospital Colorado	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	University Hospital Cleveland Medical Center/Case Western Reserve University	Cleveland	Ohio
United States	Icahn School of Medicine	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  Canada,  France,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants with Adverse Events and Serious Adverse Events		Up to 416 weeks
Secondary	Change from Baseline Over Time in the Ureagenesis Rate	Sodium acetate is used as a tracer to measure the rate of ureagenesis	Baseline (average of Screening and Day 1) up to 416 weeks following DTX301 administration
Secondary	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

External Links

•   Ultragenyx Ornithine Transcarbamylase (OTC) Deficiency Research Study Opportunity