New study tracks rare brain disease in children to speed up treatment development
NCT ID NCT05659901
First seen Jan 11, 2026 · Last updated May 24, 2026 · Updated 26 times
Summary
This study follows 32 boys and young men with Pelizaeus-Merzbacher disease, a rare genetic brain disorder, to measure how the disease changes over time. Researchers will collect spinal fluid, blood, and brain scans, and track movement, thinking, and daily function. The goal is to find reliable markers of disease progression that can be used in future treatment trials.
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 PELIZAEUS-MERZBACHER DISEASE are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Ionis Investigative Site
RECRUITINGAtlanta, Georgia, 30342, United States
-
Ionis Investigative Site
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
-
Ionis Investigative Site
ACTIVE_NOT_RECRUITINGClermont-Ferrand, France
-
Ionis Investigative Site
ACTIVE_NOT_RECRUITINGLe Kremlin-Bicêtre, 94270, France
-
Ionis Investigative Site
ACTIVE_NOT_RECRUITINGGöttingen, Germany
-
Ionis Investigative Site
RECRUITINGTel Aviv, 6423906, Israel
-
Ionis Investigative Site
RECRUITINGMilan, 20154, Italy
-
Ionis Investigative Site
RECRUITINGAmsterdam, 1081 HV, Netherlands
-
Ionis Investigative Site
RECRUITINGLeeds, LS1 3EX, United Kingdom
Conditions
Explore the condition pages connected to this study.