Experimental drug offers hope for rare brain disorder
NCT ID NCT04849741
Summary
This study is testing whether a new drug called ION373 can help improve or stabilize movement and motor function in people with Alexander disease, a rare genetic brain disorder. About 54 patients aged 2-65 will receive either the drug or a placebo for 60 weeks, followed by the drug for everyone. The main goal is to see if the drug helps patients walk better and manage their symptoms.
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 ALEXANDER DISEASE 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
-
Amsterdam Universitair Medische Centra - Academisch Medisch Centrum
Amsterdam, North Holland, 1105 AZ, Netherlands
-
Children's Hospital of Atlanta
Atlanta, Georgia, 30329, United States
-
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
-
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH, United Kingdom
-
Lucile Packard Children's Hospital Stanford
Palo Alto, California, 94304, United States
-
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
-
McGill University Health Centre
Montreal, Quebec, H3A 0G4, Canada
-
Murdoch Children's Research Institute
Parkville, Victoria, 3052, Australia
-
National Center of Neurology and Psychiatry
Tokyo, Kodaira-shi, 187-8551, Japan
-
Ospedale Pediatrico Bambino Gesù
Roma, 165, Italy
-
Ospedale dei Bambini Vittore Buzzi
Milan, 20154, Italy
-
Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906, Israel
-
University College London Hospitals NHS Foundation Trust
London, WC1N 3BG, United Kingdom
Conditions
Explore the condition pages connected to this study.