Natural History Study of Batten Disease (NCT04644549) | Clinical Trial Compass
TerminatedNot Applicable
Natural History Study of Batten Disease
Stopped: study terminated following strategic portfolio and R\&D alignment
United States10 participantsStarted 2021-04-27
Plain-language 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.
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Assess the change over time in rating as determined using the Unified Batten Disease Rating Scale (UBDRS).
Timeframe: 10 years
2
Assess the change over time in rating as determined using the Hamburg Scale.
Timeframe: 10 years
3
Assess the change over time in cognitive function using Mullen Scales of Early Learning (for children up to 60 months old).
Timeframe: 10 years
4
Assess the change over time in cognitive function using WPPSI-IV (for children up to 7 years 7 months old).
Timeframe: 10 years
5
Assess the change over time in cognitive function using WISC-V (for patients older than 6 years old).
Timeframe: 10 years
6
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