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NCT04470713 · ClinicalTrials.gov registry record
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
A clinical trial of GM1 Gangliosidosis and Tay-Sachs Disease, sponsored by Idorsia Pharmaceuticals.
- Completed
- Registry status
- 226
- Enrollment target
- 17
- Study locations
NCT04470713: Completed study of GM1 Gangliosidosis and Tay-Sachs Disease, sponsored by Idorsia Pharmaceuticals.
NCT04470713 is a study of GM1 Gangliosidosis and Tay-Sachs Disease that has completed, run by Idorsia Pharmaceuticals. The registered enrollment target is 226 participants, above the 69-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (228% higher). The trial reports 17 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04470713, a study of GM1 Gangliosidosis and Tay-Sachs Disease, has completed, sponsored by Idorsia Pharmaceuticals.
- COMPLETED
- Registry status
- 226 participants
- Enrollment target
- 17
- Study locations
Study Summary
This study is being conducted to better understand the natural course of GM1 gangliosidosis, GM2 gangliosidoses and Gaucher disease Type 2 (GD2). Information is planned to be gathered on at least 180 patients with GM1 gangliosidosis, GM2 gangliosidoses, and Gaucher Disease type 2. Retrospective data collection is planned for at least 150 deceased patients (Group A). Group B is for patients alive at the time of enrollment. In Group B it is planned to prospectively collect more comprehensive data from at least 30 patients. The purpose of this study is to collect relevant information for a adequate design of a potential subsequent research program in these diseases. In this study no therapy is being offered.
Conditions Studied
Study Locations (17)
Other
- UCL Cliniques Universitaires Saint-Luc - Brussels
- Hospital de Clínicas de Porto Alegre - HCPA - Porto Alegre
- AP-HP - Hôpitaux Universitaires Est Parisien - Paris
- SphinCS GmbH - Höchheim
- Azienda Ospedaliero Universitaria Meyer - Florence
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta - Milan
- Centro Hospitalar Universitario Lisboa Norte, EPE - Lisbon
- Centro Universitario Hospitalar de São João, EPE - Porto
- Hospital Sant Joan de Deu - Barcelona
- Quirónsalud - Zaragoza
- Universitätsspital Bern Inselspital - Bern
- Universitäts-Kinderspital Zürich - Zurich
- University Hospitals Birmingham NHS Foundation Trust - Birmingham
- Great Ormond Street Hospital for Children NHS Found. Trust - London
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Minnesota
- Mayo Clinic - Rochester - Rochester
Virginia
- Lysosomal and Rare Disorders Research and Treatment Center - Fairfax
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 226 participants |
| Start Date | 2019-07-31 |
| Est. Completion | 2021-10-30 |
What the finished NCT04470713 record still lists
NCT04470713 is an observational study that tracks outcomes without assigning an intervention. The registered 226 participants enrollment target is mid-sized for trials with a published cap, above the 69-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (228% higher).
The record links to 6 conditions, with GM1 Gangliosidosis appearing as the primary indexed condition, and to 0 interventions.
NCT04470713 lists 17 locations in 4 states (Other, Illinois, Minnesota).
Frequently Asked Questions
What is clinical trial NCT04470713 about?
NCT04470713 is a clinical study titled "Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2". This study is being conducted to better understand the natural course of GM1 gangliosidosis, GM2 gangliosidoses and Gaucher disease Type 2 (GD2). Information is planned to be gathered on at least 180 patients with GM1 gangliosidosis, GM2 gangliosidoses, and Gaucher Disease type 2. Retrospective data...
What is the current status of trial NCT04470713?
This trial is currently completed. The enrollment target is 226 participants. The study started on 2019-07-31. Estimated completion is 2021-10-30.
What conditions does trial NCT04470713 study?
This clinical trial studies the following conditions: GM1 Gangliosidosis, Tay-Sachs Disease, Sandhoff Disease, GM2 Gangliosidosis, Gaucher Disease, Type 2.
Who is sponsoring clinical trial NCT04470713?
This trial is sponsored by Idorsia Pharmaceuticals, which has 34 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04470713 being conducted?
This trial has 17 study locations across Illinois, Minnesota, Virginia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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