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.
Disclaimer
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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.
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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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
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Locations
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Texas Children Hospital
Houston, Texas, 77030, United States