Natural History Study of Batten Disease

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Total Enrollment

10 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-04-27

Study Completion Date

2022-03-11

Brief Summary

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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.

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Detailed Description

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Conditions

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Batten Disease Neuronal Ceroid Lipofuscinosis CLN6 Neuronal Ceroid Lipofuscinosis CLN3

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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Subjects with CLN6 Batten disease

No interventions assigned to this group

Subjects with juvenile CLN3 Batten disease

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* Subject has or had a diagnosis of CLN6 or CLN3 Batten disease that has been confirmed by genotyping (documented presence of a variant on both gene alleles). Confirmation of genotyping will be performed prior to enrollment.
* Subject (or legally authorized representative) has provided written informed consent (or assent) and authorization for use and disclosure of personal health information or research related health information
* Subjects may enroll starting from birth

Exclusion Criteria

* Subject has or had experienced another illness that is known to cause cognitive decline (eg, trauma, meningitis, hemorrhage)
* Subject received or receives an investigational gene therapy

Eligible Sex

ALL

Accepts Healthy Volunteers

No