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NCT04041102 · ClinicalTrials.gov registry record
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
A clinical trial of GM1 Gangliosidosis, sponsored by University of Pennsylvania.
- Completed
- Registry status
- 31
- Enrollment target
- 6
- Study locations
NCT04041102: Completed study of GM1 Gangliosidosis, sponsored by University of Pennsylvania.
NCT04041102 is a study of GM1 Gangliosidosis that has completed, run by University of Pennsylvania. The registered enrollment target is 31 participants, below the 102-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (70% lower). The trial reports 6 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04041102, a study of GM1 Gangliosidosis, has completed, sponsored by University of Pennsylvania.
- COMPLETED
- Registry status
- 31 participants
- Enrollment target
- 6
- Study locations
Study Summary
Owing to the rarity, severity, speed of progression and fatal prognosis of infantile and juvenile GM1, there is a limited understanding of overall disease progression and meaningful outcome measures. This study aims to build a natural history data set through collection of a number of clinical, imaging, and laboratory assessments that may be specific predictors of GM1 disease progression and clinical outcome. Having a GM1 natural history data set can inform potential efficacy endpoints and biomarkers for future clinical trials. This natural history study will follow up to 40 subjects diagnosed with GM1 gangliosidosis (up to 20 infantile (Type 1) and 20 late infantile/juvenile (Type 2)) for up to 3 years. Visits will be conducted every 6 months, during which several procedures will be performed and the data recorded in order to learn about the natural course of the disease, including changes in clinical and neurological assessments and electrophysiologic, imaging and biofluid biomarkers. Study procedures include: physical \& neurological exam, blood \& urine sample collection, questionnaires \& assessments of development, seizure diary, ECHO, ECG, x-ray and ultrasound (if MRI not performed), EEG and genetic testing (if not already done). The following procedures are subject to local/institutional policies and the medical discretion of the Study Physician: MRI, lumbar puncture (spinal tap) and General anesthesia/sedation (for MRI and LP).
Primary Outcome
Survival will be evaluated at the baseline visit and each subsequent visit for 3 years. This outcome measure is considered a primary outcome measure for the infantile (Type 1) GM1 population and a secondary outcome measure for the juvenile (Type 2) GM1 population.
Conditions Studied
Study Locations (6)
Other
- Hospital de Clínicas de Porto Alegre - Porto Alegre
- Gazi University - Ankara
- UCL Great Ormond Street Institute of Child Health - London
California
- UCSF Benioff Children's Hospital Oakland - Oakland
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Quebec
- Montreal Children's Hospital Research Institute - McGill University - Montreal
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 31 participants |
| Start Date | 2020-06-12 |
| Est. Completion | 2024-05-01 |
What the finished NCT04041102 record still lists
NCT04041102 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 31 participants, a relatively small participant target, below the 102-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (70% lower).
The record links to 1 condition, with GM1 Gangliosidosis appearing as the primary indexed condition, and to 0 interventions.
NCT04041102 lists 6 locations in 4 states (Other, California, Pennsylvania).
Frequently Asked Questions
What is clinical trial NCT04041102 about?
NCT04041102 is a clinical study titled "Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients". Owing to the rarity, severity, speed of progression and fatal prognosis of infantile and juvenile GM1, there is a limited understanding of overall disease progression and meaningful outcome measures. This study aims to build a natural history data set through collection of a number of clinical, imag...
What is the current status of trial NCT04041102?
This trial is currently completed. The enrollment target is 31 participants. The study started on 2020-06-12. Estimated completion is 2024-05-01.
What conditions does trial NCT04041102 study?
This clinical trial studies the following conditions: GM1 Gangliosidosis.
Who is sponsoring clinical trial NCT04041102?
This trial is sponsored by University of Pennsylvania, which has 1,423 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04041102 being conducted?
This trial has 6 study locations across California, Pennsylvania, Quebec. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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