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Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency | Clinical Trials | Clareo Health

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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

NCT03636438•Ultragenyx Pharmaceutical Inc

View on ClinicalTrials.gov

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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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.

Locations (9)

The Children's Hospital Colorado

Aurora, Colorado, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Icahn School of Medicine

New York, New York, United States

University Hospital Cleveland Medical Center/Case Western Reserve University

Cleveland, Ohio, United States

M.A.G.I.C. Clinic

Calgary, Alberta, Canada

Hopital Femme Mere Enfant

Bron, Rhone, France

Hospital Clinico Universitario de Santiago

Santiago de Compostela, Coruna, Spain

Hospital Universitario de Cruces. Servicio de Pediatria

Barakaldo, Vizcaya, Spain

Queen Elizabeth Hospital, Department of Endocrinology

Birmingham, United Kingdom

Key Dates

Start Date

August 30, 2018

Primary Completion Date

December 1, 2029

Completion Date

December 1, 2029

Last Updated

February 19, 2026

Enrollment

ACTUAL participants

Conditions

Ornithine Transcarbamylase (OTC) Deficiency

Interventions

No Intervention

OTHER