CompletedNot Applicable

Interviews and Video Capture in Patients With GM1 Gangliosidosis

United States25 participantsStarted 2020-04-20

Plain-language 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 not always well-suited to capture all the disease features and other metrics that have an impact on the patient and family. To address the methodological challenges of this small, heterogeneous population, this study will collect patient-specific home-based video data and qualitative interviews with caregivers.

Who can participate

SexALL

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Inclusion criteria

✓. 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

What they're measuring

Scheduled Video Capture

Timeframe: up to 24 months

Trial details

NCT IDNCT04310163

SponsorLYSOGENE

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2023-05-05

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