New hope for rare genetic disorder: ION440 trial launches
NCT ID NCT06430385
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 26 times
Summary
This study tests a new medicine called ION440 for people with MECP2 duplication syndrome, a rare genetic condition that causes intellectual disability and movement problems. The drug is given via a spinal injection to see if it is safe and how the body processes it. About 48 males aged 2 to 65 will take part in this early-stage trial.
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 METHYL CPG BINDING PROTEIN 2 (MECP2) DUPLICATION 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
-
Baylor College of Medicine
RECRUITINGHouston, Texas, 77030, United States
-
Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
-
CHU Dijon Bourgogne
RECRUITINGDijon, 21079, France
-
Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
-
Gillette Children's Specialty Healthcare
RECRUITINGSaint Paul, Minnesota, 55101, United States
-
Hospital Saint Joan de Deu
RECRUITINGBarcelona, 8950, Spain
-
Kennedy Krieger
RECRUITINGBaltimore, Maryland, 21205, United States
-
Kepler University Hospital
RECRUITINGLinz, 4040, Austria
-
Rady Children's Hospital
RECRUITINGSan Diego, California, 92123, United States
-
University of Colorado Hopsital - Anschutz Medical Campus
RECRUITINGAurora, Colorado, 80045, United States
-
Vanderbilt University Medical Center
RECRUITINGNashville, Tennessee, 37232, United States
Conditions
Explore the condition pages connected to this study.