Status:
COMPLETED
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
Lead Sponsor:
Idorsia Pharmaceuticals Ltd.
Conditions:
GM1 Gangliosidosis
GM2 Gangliosidosis
Eligibility:
All Genders
Brief 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 ...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- Patient with either GM1 gangliosidosis, GM2 gangliosidoses (Tay-Sachs, Sandhoff, or AB Variant), or Gaucher Disease Type 2.
- Diagnosis confirmed by either biochemical (enzyme activity) or genetic testing, or both.
- Date of birth on or after 1 January 2000.
- Onset of first neurological symptom within 24 months of age.
- Informed consent of parent or legal guardian as required by local law.
Exclusion
Key Trial Info
Start Date :
July 31 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
October 30 2021
Estimated Enrollment :
226 Patients enrolled
Trial Details
Trial ID
NCT04470713
Start Date
July 31 2019
End Date
October 30 2021
Last Update
November 8 2021
Active Locations (17)
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1
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
2
Mayo Clinic - Rochester
Rochester, Minnesota, United States, 55905
3
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States, 22030
4
UCL Cliniques Universitaires Saint-Luc
Brussels, Belgium, 1020