Interviews and Video Capture in Patients With GM1 Gangliosidosis

GM1 Gangliosidosis Clinical Trial

Official title:

Natural History Study Using Interview and Video Capture of Infantile and Juvenile GM1 Gangliosidosis (GM1)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04310163
• Other study ID #: CAS-LYS003-01
• Status: Completed
• Start date: April 20, 2020
• Est. completion date: May 5, 2023

Study information

• Verified date: June 2023
• Source: LYSOGENE
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 and follow-up timepoints throughout the study (3, 6, 12, 18, and 24 months) and/or submit videos taken in the past through a secure smart phone mobile application. The video assessments focus on several hallmarks of GM1 gangliosidosis progression. Caregivers participate in qualitative interviews to provide context for the videos and discuss any changes they observe during the study. Activity videos will be evaluated by expert clinicians using both Clinical Global Impression of Severity (CGI-S) and Clinical Global Impression of Change (CGI-C) scales. The caregiver interviews and clinician-rated activities will inform the patient-specific disease trajectories for each hallmark. There is no treatment or intervention associated with this study.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: May 5, 2023
• Est. primary completion date: May 5, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

Be or have been the parent, legal guardian, or caretaker of a patient with GM1

gangliosidosis with:

1. Early infantile GM1 gangliosidosis

2. Late infantile GM1 gangliosidosis

3. Juvenile GM1 gangliosidosis who can walk with assistance or possesses past videos of when child could walk with assistance

4. 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 Criteria: GM1 gangliosidosis patient that the caregiver cares for is being treated with any experimental medication in a clinical trial setting.

Related Conditions & MeSH terms

• Gangliosidoses
• Gangliosidosis, GM1
• GM1 Gangliosidosis

Intervention

Other:
• Natural history
Parent interview and video capture

Locations

Country	Name	City	State
United States	Casimir Trials	Plymouth	Massachusetts

Sponsors (3)

Lead Sponsor	Collaborator
LYSOGENE	Casimir, LLC, Cure GM1 Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Scheduled Video Capture	Caregivers will record GM1 gangliosidosis participants doing specific activities of daily living. A list of standardized activities will be provided at baseline and the caregiver will select the activities that are relevant to the GM1 gangliosidosis participant. The activities include: gross motor skills, fine motor skills, caregiver interaction, communication, self-care and visual tracking.	up to 24 months
Secondary	Unscheduled Video Capture	In addition to the scheduled video captures, spontaneously-captured videos may also be submitted by the caregivers at any time. These spontaneous videos should demonstrate any behavior or ability that the caregivers consider to be a meaningful change for the participant.	up to 24 months
Secondary	Caregiver Interviews	Casimir study staff will conduct video interviews with the caregivers at baseline and follow-up timepoints .	up to 24 months