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Trial record 1 of 10 for:    Recruiting, Not yet recruiting, Active, not recruiting Studies | Gm1 gangliosidosis
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Interviews and Video Capture in Patients With GM1 Gangliosidosis

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ClinicalTrials.gov Identifier: NCT04310163
Recruitment Status : Recruiting
First Posted : March 17, 2020
Last Update Posted : October 12, 2020
Sponsor:
Collaborators:
Casimir, LLC
Cure GM1 Foundation
Information provided by (Responsible Party):
LYSOGENE

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 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.

Condition or disease Intervention/treatment
GM1 Gangliosidosis Other: Natural history

Detailed Description:
This is a natural history study, for up to 2 years, during which parents or guardians of a GM1 child 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 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.

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Study Type : Observational
Estimated Enrollment : 30 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Natural History Study Using Interview and Video Capture of Infantile and Juvenile GM1 Gangliosidosis (GM1)
Actual Study Start Date : April 20, 2020
Estimated Primary Completion Date : May 2023
Estimated Study Completion Date : May 2023



Intervention Details:
  • Other: Natural history
    Parent interview and video capture


Primary Outcome Measures :
  1. Scheduled Video Capture [ Time Frame: up to 24 months ]
    Caregivers will record GM1 gangliosidosis participants doing some standardized activities. 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.


Secondary Outcome Measures :
  1. Unscheduled Video Capture [ Time Frame: up to 24 months ]
    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.

  2. Caregiver Interviews [ Time Frame: up to 24 months ]
    Casimir study staff will conduct video interviews with the caregivers at baseline and follow-up timepoints .



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Study population will consist of children or adolescents
Criteria

Inclusion Criteria:

Be the parent or legal guardian of a patient with GM1 gangliosidosis who meets the self-reported criteria listed below for each sub-type or be in possession of past videos of when the child met the criteria:

  • A patient with early infantile GM1 gangliosidosis who can lift his/her head
  • A patient with late infantile GM1 gangliosidosis who can sit with hand support
  • A patient with juvenile GM1 gangliosidosis who can walk with assistance

Exclusion Criteria:

Parent or legal guardian of a patient with GM1 gangliosidosis who is participating in any ongoing clinical trial at the time of study enrollment or enrolls in a clinical trial during this study.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04310163


Contacts
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Contact: Casimir Trials, PhD, MPH 1 800 378 9569 GM1@casimirtrials.com

Locations
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United States, Massachusetts
Casimir Trials Recruiting
Plymouth, Massachusetts, United States, 02360
Contact: Marielle Contesse, PhD, MPH       GM1@casimirtrials.com   
Sponsors and Collaborators
LYSOGENE
Casimir, LLC
Cure GM1 Foundation
Investigators
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Principal Investigator: Mindy Leffler, MEd Casimir Trials
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Responsible Party: LYSOGENE
ClinicalTrials.gov Identifier: NCT04310163    
Other Study ID Numbers: CAS-LYS003-02
First Posted: March 17, 2020    Key Record Dates
Last Update Posted: October 12, 2020
Last Verified: October 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by LYSOGENE:
GM1 gangliosidosis
Landing disease
Lysosomal Storage Disease
Additional relevant MeSH terms:
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Gangliosidoses
Gangliosidosis, GM1
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders