Clinical Trials in the USA / 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 USA tracker
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
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)
- Number of Participants with Adverse Events and Serious Adverse Events — Up to 416 weeks
Trial sites (9)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
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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 the USA