Rare disease study tracks symptoms in just 5 patients
NCT ID NCT04098211
First seen Apr 23, 2026 · Last updated May 24, 2026 · Updated 7 times
Summary
This study follows a small group of people with a rare, late-onset form of TPP1 deficiency (also called CLN2 disease) to learn how their symptoms change over time. Researchers will use tests like brain scans, eye exams, and movement assessments to gather information. The goal is to better understand the disease, not to test a new treatment.
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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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Contacts and locations
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Locations
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Children's Hospital of Orange County
Orange, California, 92868, United States
Conditions
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