Natural History Study of Batten Disease
NCT04644549 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 10
Last updated 2025-07-30
Summary
This is a multicenter, international, study to assess the natural history data from people with Batten disease by collecting both retrospective and prospective information about the motor, behavioral and functional capabilities of patients. The study initially has 2 cohorts. Cohort 1 (n ≈ 75) includes subjects with CLN6 Batten disease. Cohort 2 (n ≈ 120) includes subjects with juvenile CLN3 Batten disease. Additional cohorts for other Batten disease subtypes may be added in the future.
Conditions
- Batten Disease
- Neuronal Ceroid Lipofuscinosis CLN6
- Neuronal Ceroid Lipofuscinosis CLN3
Sponsors & Collaborators
-
Emily de los Reyes
lead OTHER
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2021-04-27
- Primary Completion
- 2022-03-11
- Completion
- 2022-03-11
Countries
- United States
Study Locations
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