A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term

Phase 1

• Conditions: Ornithine transcarbamylase deficiency; MedDRA version: 20.0Level: LLTClassification code 10071107Term: Ornithine transcarbamylase deficiencySystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2018-000156-18-FR
• Lead Sponsor: ltragenyx Pharmaceutical, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-01-31
• Last Update Posted: 16 November 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

1. Completed the Week 52 visit in Study 301OTC01. Note that the Day 0 visit of Study 301OTC02 may coincide with the Week 52 visit of 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.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 17
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified