Scientists track Long-Term safety of One-Time gene fix for rare disorder
NCT ID NCT03636438
Summary
This study is tracking the long-term safety and effectiveness of a one-time gene therapy for adults with late-onset OTC deficiency, a rare genetic disorder that affects the body's ability to process protein. It follows 11 adults who previously received the experimental treatment in an earlier trial. The main goal is to monitor for any side effects over a long period and see if the treatment continues to help control the disease.
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.