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NCT04310163 · ClinicalTrials.gov registry record
Interviews and Video Capture in Patients With GM1 Gangliosidosis
A clinical trial of GM1 Gangliosidosis, sponsored by LYSOGENE.
- Completed
- Registry status
- 25
- Enrollment target
- 1
- Study location
NCT04310163: Completed study of GM1 Gangliosidosis, sponsored by LYSOGENE.
NCT04310163 is a study of GM1 Gangliosidosis that has completed, run by LYSOGENE. The registered enrollment target is 25 participants, below the 103-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (76% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04310163, a study of GM1 Gangliosidosis, has completed, sponsored by LYSOGENE.
- COMPLETED
- Registry status
- 25 participants
- Enrollment target
- 1
- Study location
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.
Primary Outcome
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.
Conditions Studied
Interventions
- OTHER Natural history
Study Locations (1)
Massachusetts
- Casimir Trials - Plymouth
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 25 participants |
| Start Date | 2020-04-20 |
| Est. Completion | 2023-05-05 |
What the finished NCT04310163 record still lists
NCT04310163 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 25 participants, a relatively small participant target, below the 103-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (76% lower).
The record links to 1 condition, with GM1 Gangliosidosis appearing as the primary indexed condition, and to 1 intervention - of which Natural history is the first listed.
NCT04310163 reports a single indexed study location in Massachusetts.
Frequently Asked Questions
What is clinical trial NCT04310163 about?
NCT04310163 is a clinical study titled "Interviews and Video Capture in Patients With GM1 Gangliosidosis". 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 th...
What is the current status of trial NCT04310163?
This trial is currently completed. The enrollment target is 25 participants. The study started on 2020-04-20. Estimated completion is 2023-05-05.
What conditions does trial NCT04310163 study?
This clinical trial studies the following conditions: GM1 Gangliosidosis.
What interventions are being tested in trial NCT04310163?
The interventions under investigation include: Natural history (OTHER).
Who is sponsoring clinical trial NCT04310163?
This trial is sponsored by LYSOGENE, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04310163 being conducted?
This trial has 1 study location across Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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