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Clinical Trials in France / NCT03636438
Active, not recruiting Observational

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

NCT03636438 · tracked via the Priya Life Science France tracker
Sponsor
Ultragenyx Pharmaceutical Inc
Phase
Observational
Started
2018-08-30
Last updated
2026-06-01

Condition(s) studied

Ornithine Transcarbamylase (OTC) Deficiency

Investigational drug(s) / intervention(s)

No Intervention

No Intervention: No Intervention

Study summary

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

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (9)

FacilityCityRegionStatus
The Children's Hospital Colorado Aurora Colorado
Boston Children's Hospital Boston Massachusetts
Icahn School of Medicine New York New York
University Hospital Cleveland Medical Center/Case Western Reserve University Cleveland Ohio
M.A.G.I.C. Clinic Calgary Alberta
Hopital Femme Mere Enfant Bron Rhone
Hospital Clinico Universitario de Santiago Santiago de Compostela Coruna
Hospital Universitario de Cruces. Servicio de Pediatria Barakaldo Vizcaya
Queen Elizabeth Hospital, Department of Endocrinology Birmingham United Kingdom

More Ultragenyx Pharmaceutical Inc trials in France

Long-term Extension of GTX-102 in Angelman Syndrome Active, not recruiting
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency Active, not recruiting
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) Active, not recruiting
Setrusumab vs Placebo for Osteogenesis Imperfecta Active, not recruiting
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03636438 on ClinicalTrials.gov ↗ ← All trials in France