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.

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.

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Conditions

The condition(s) this trial relates to.

juvenile neuronal ceroid lipofuscinosis neuronal ceroid lipofuscinosis neuronal ceroid lipofuscinosis 3 Neuronal Ceroid-Lipofuscinoses

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

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

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

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