Gene therapy for rare liver disease: Long-Term safety check
NCT ID NCT03636438
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study follows 11 adults with late-onset OTC deficiency who received a single dose of gene therapy (DTX301) in an earlier trial. Researchers are checking long-term safety and how well the body makes urea and controls ammonia levels. The goal is to see if the treatment remains safe over time.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
-
Hopital Femme Mere Enfant
Bron, Rhone, 69677, France
-
Hospital Clinico Universitario de Santiago
Santiago de Compostela, Coruna, 15706, Spain
-
Hospital Universitario de Cruces. Servicio de Pediatria
Barakaldo, Vizcaya, 48903, Spain
-
Icahn School of Medicine
New York, New York, 10029, United States
-
M.A.G.I.C. Clinic
Calgary, Alberta, T2E 7Z7, Canada
-
Queen Elizabeth Hospital, Department of Endocrinology
Birmingham, B15 2TH, United Kingdom
-
The Children's Hospital Colorado
Aurora, Colorado, 80045, United States
-
University Hospital Cleveland Medical Center/Case Western Reserve University
Cleveland, Ohio, 44106, United States