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Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

NCT04470713 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 226

Last updated 2021-11-08

No results posted yet for this 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

  • GM1 Gangliosidosis
  • GM2 Gangliosidosis
  • Gaucher Disease, Type 2
  • Tay-Sachs Disease
  • AB Variant Gangliosidosis GM2
  • Sandhoff Disease

Sponsors & Collaborators

  • Idorsia Pharmaceuticals Ltd.

    lead INDUSTRY

Principal Investigators

  • Clinical Trials · Idorsia Pharmaceuticals Ltd.

Eligibility

Min Age
0 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-07-31
Primary Completion
2021-10-30
Completion
2021-10-30

Countries

  • United States
  • Belgium
  • Brazil
  • France
  • Germany
  • Italy
  • Portugal
  • Spain
  • Switzerland
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04470713 on ClinicalTrials.gov