Investigations of Juvenile Neuronal Ceroid Lipofuscinosis
Recruiting
Observational Study
Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)
Batten Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 1 Week – 100
- Sex
- Any
- Study type
- Observational
- Participants needed
- 300 (estimated)
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · NIH
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About This Trial
Background:
CLN3, or Batten disease, is a genetic disorder. This deadly disease leads to decline of brain and nervous system functions. Symptoms of CLN3 typically occur between 4 and 7 years of age. They include changes in how a person sees, thinks, and moves. CLN3 can also cause seizures. No effective treatments for the disease are yet known. There is limited testing of potential therapies. Rese…
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA:
For the Main and Sub-Studies, participants \> 1 week of age, of all genders, demographics, geographic locations, and disease severity will be recruited in order to obtain cross-sectional representation of CLN3-related conditions (Main and Sub-Studies) or all NCLs (Sub-Study B)…
Contacts