New hope for rare brain disease: first drug trial in boys

NCT ID NCT06150716

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

First seen Jan 08, 2026 · Last updated May 21, 2026 · Updated 20 times

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 see if the drug is safe and tolerable when given via spinal injection. Researchers will also measure how the drug moves through the body and its effects on symptoms.

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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.

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Contacts and locations

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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

Conditions

Explore the condition pages connected to this study.

PELIZAEUS-MERZBACHER DISEASE