One-Time brain injection aims to halt devastating genetic disorder in girls
NCT ID NCT05898620
Summary
This study is testing a one-time gene therapy called NGN-401 for girls with Rett syndrome, a severe genetic disorder that impairs brain development. The therapy delivers a working copy of a missing gene directly to the brain. Researchers will follow participants for 3 years to see if it safely helps them regain developmental skills and improve their condition.
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 RETT SYNDROME 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
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact Email: •••••@•••••
-
Children's Hospital of Philadelphia
NOT_YET_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
-
Kennedy Krieger Institute
RECRUITINGBaltimore, Maryland, 21205, United States
Contact Email: •••••@•••••
-
Manchester University NHS Foundation Trust
TERMINATEDManchester, M13 9WL, United Kingdom
-
Montefiore Medical Center
RECRUITINGNew York, New York, 10467, United States
Contact Email: •••••@•••••
-
Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
Contact Email: •••••@•••••
-
Nicklaus Children's Hospital Research Institute
RECRUITINGMiami, Florida, 33155, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Royal Hospital for Children and Young People
TERMINATEDEdinburgh, EH16 4TJ, United Kingdom
-
Rush University Medical Center
RECRUITINGChicago, Illinois, 60612, United States
Contact Email: •••••@•••••
-
Texas Children's Hospital
RECRUITINGHouston, Texas, 77030, United States
Contact Email: •••••@•••••
-
The Children's Hospital at Westmead
ACTIVE_NOT_RECRUITINGSydney, New South Wales, Australia
-
UCSF Benioff Children's Hospital Oakland
RECRUITINGOakland, California, 94609, United States
Contact Email: •••••@•••••
-
UNC at Chapel Hill
RECRUITINGChapel Hill, North Carolina, 27514, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
University of Alabama at Birmingham
RECRUITINGBirmingham, Alabama, 35233, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Vanderbilt University Medical Center
RECRUITINGNashville, Tennessee, 37232, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.