Effects of Miglustat on systemic and neurological symptoms of Sandhoff and Tay-Sachs diseases
Phase 4
Recruiting
- Conditions
- Condition 1: Sandhoff. Condition 2: Tay-Sachs.GM2 gangliosidosis
- Registration Number
- IRCT20181023041435N1
- Lead Sponsor
- Tehran University of Medical Sciences
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 30
Inclusion Criteria
Clinically and genetically confirmed cases of Sandhoff (SD)/Tay-Sachs (TSD) diseases
Enrolled patients who are referred to Myelin Disorders Clinic, Children’s Medical Center with clinically suspicion to TSD and SD
Exclusion Criteria
Diarrhea
Renal impairement
Loss of follow up
Other systemic diseases
Concomitant drug therapy which may affect neurological system function
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method