Scientists launch major study to unlock secrets of deadly batten disease
NCT ID NCT03307304
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 300 people with CLN3 Batten disease, a rare genetic disorder that causes vision loss, seizures, and decline in thinking and movement. Researchers collect samples like blood and spinal fluid to find biological markers that could be used in future treatment trials. No experimental drug is given; the goal is to better understand the disease and prepare for future therapies.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- What this could lead to
- If successful, this study could identify biological markers that help design future treatments for Batten disease.
- What could go wrong
- This is an observational study, not a treatment trial. It will not directly improve symptoms, and findings may not lead to a therapy.
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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
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Experimental gene therapy aims to halt rare childhood brain disease
- Major study tracks rare brain diseases to unlock their secrets
- Experimental drug miglustat tested for rare batten disease
- Scientists track batten disease to unlock its secrets