Stopped: study terminated following strategic portfolio and R\&D alignment
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.
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Assess the change over time in rating as determined using the Unified Batten Disease Rating Scale (UBDRS).
Timeframe: 10 years
Assess the change over time in rating as determined using the Hamburg Scale.
Timeframe: 10 years
Assess the change over time in cognitive function using Mullen Scales of Early Learning (for children up to 60 months old).
Timeframe: 10 years
Assess the change over time in cognitive function using WPPSI-IV (for children up to 7 years 7 months old).
Timeframe: 10 years
Assess the change over time in cognitive function using WISC-V (for patients older than 6 years old).
Timeframe: 10 years
Characterize the age of onset of disease, including the timing of both the loss of capacities and the emergence of disease-related signs and symptoms
Timeframe: 10 years