Status:
COMPLETED
Interviews and Video Capture in Patients With GM1 Gangliosidosis
Lead Sponsor:
LYSOGENE
Collaborating Sponsors:
Casimir, LLC
Cure GM1 Foundation
Conditions:
GM1 Gangliosidosis
Eligibility:
All Genders
Brief Summary
GM1 gangliosidosis is a rare disease for which there is a limited understanding of disease progression and meaningful outcome measures. In addition, parents report that clinic-based assessments are no...
Detailed Description
This is a natural history study, for up to 2 years, during which parents or guardians of a child with GM1 gangliosidosis collect video data of patients doing specific daily life activities at baseline...
Eligibility Criteria
Inclusion
- Be or have been the parent, legal guardian, or caretaker of a patient with GM1 gangliosidosis with:
- Early infantile GM1 gangliosidosis
- Late infantile GM1 gangliosidosis
- Juvenile GM1 gangliosidosis who can walk with assistance or possesses past videos of when child could walk with assistance
- Early or late infantile GM1 gangliosidosis who has passed away, but is in possession of videos documenting the onset and evolution of disease hallmarks of GM1 gangliosidosis
Exclusion
- GM1 gangliosidosis patient that the caregiver cares for is being treated with any experimental medication in a clinical trial setting.
Key Trial Info
Start Date :
April 20 2020
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
May 5 2023
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT04310163
Start Date
April 20 2020
End Date
May 5 2023
Last Update
June 8 2023
Active Locations (1)
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1
Casimir Trials
Plymouth, Massachusetts, United States, 02360