New drug trial offers hope for rare brain disease in boys

NCT ID NCT06150716

Recruiting now Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage study tests a new medicine called ION356 in 24 boys aged 2 to 17 with Pelizaeus-Merzbacher disease, a rare genetic brain disorder. The main goal is to check if the drug is safe and how the body processes it. The drug is given through a spinal injection to target the brain directly.

Disclaimer Read more

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.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Pelizaeus-Merzbacher spectrum disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PELIZAEUS-MERZBACHER DISEASE

Contacts and locations

Show contact details

Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Ionis Investigative Site
RECRUITING

Atlanta, Georgia, 30342, United States
Ionis Investigative Site
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Ionis Investigative Site
RECRUITING

Salt Lake City, Utah, 84113, United States
Ionis Investigative Site
RECRUITING

Le Kremlin-Bicêtre, 94270, France
Ionis Investigative Site
RECRUITING

Göttingen, 37075, Germany
Ionis Investigative Site
RECRUITING

Tokyo, 187-8551, Japan
Ionis Investigative Site
RECRUITING

Amsterdam, 1105AZ, Netherlands