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.
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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

An N Dang Do, M.D.

(301) 496-8849

an.dangdo@nih.gov

CONTACT