Experimental drug miglustat tested for rare batten disease
NCT ID NCT05174039
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This study tested the drug miglustat in 6 adults aged 17 and older with CLN3 Batten disease, a rare genetic disorder that causes nerve damage. The goal was to see if the drug is safe and to find the best dose over 2 years. Researchers tracked side effects and how the drug moved through the body.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- miglustat
- What this could lead to
- If it works, this could point toward a treatment that slows or manages CLN3 Batten disease in older patients.
- What could go wrong
- This was a very small, early-phase trial with only 6 people, so results may not apply widely. The drug may cause side effects and may not improve the disease.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for BATTEN DISEASE are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Texas Children Hospital
Houston, Texas, 77030, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Experimental gene therapy aims to halt rare fatal brain disease in children
- Thousands join fight against blindness by sharing their stories
- Experimental gene therapy aims to halt rare childhood brain disease
- Major study tracks rare brain diseases to unlock their secrets
- Scientists launch major study to unlock secrets of deadly batten disease
- Scientists track batten disease to unlock its secrets