One-Shot gene therapy tested for Long-Term safety in rare metabolic disorder
NCT ID NCT03636438
First seen Nov 17, 2025 · Last updated Apr 12, 2026 · Updated 19 times
Summary
This study is tracking the long-term safety and effectiveness of a single-dose gene therapy for adults with a rare, inherited metabolic disorder called late-onset OTC deficiency. The therapy aims to deliver a working copy of a missing liver enzyme to help the body process ammonia. Only 11 adults who received the therapy in a previous study are being monitored for side effects and changes in their ammonia levels 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
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 7Z4, 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
Conditions
Explore the condition pages connected to this study.