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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 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.
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).
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04041102
The ClinicalTrials.gov registry entry for NCT04041102 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 31 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 102-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (70% lower). The listed sponsor is University of Pennsylvania, which has 1,423 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with GM1 Gangliosidosis appearing as the primary indexed condition, and to 0 interventions.
NCT04041102 reports 6 study locations spanning 4 distinct geographic areas - top geographies include 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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Read our methodology - how this data is sourced, computed, and verified.
Related
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