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Interviews and Video Capture in Patients With GM1 Gangliosidosis

NCT04310163 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 25

Last updated 2023-06-08

No results posted yet for this study

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.

Conditions

  • GM1 Gangliosidosis

Interventions

OTHER

Natural history

Parent interview and video capture

Sponsors & Collaborators

  • Casimir, LLC

    collaborator UNKNOWN

  • Cure GM1 Foundation

    collaborator UNKNOWN

  • LYSOGENE

    lead INDUSTRY

Principal Investigators

  • Mindy Leffler, MEd · Casimir Trials

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-04-20
Primary Completion
2023-05-05
Completion
2023-05-05

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04310163 on ClinicalTrials.gov