Status:
TERMINATED
Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)
Lead Sponsor:
Tehran University of Medical Sciences
Collaborating Sponsors:
Mashhad University of Medical Sciences
Kashan University of Medical Sciences
Conditions:
GM2 Gangliosidosis
Supportive Care
Eligibility:
All Genders
6-24 years
Phase:
PHASE3
Brief Summary
GM2 gangliosidosis is an autosomal recessive subtype of Lysosomal Storage Diseases in which, Hexosaminidase A-B deficiency is caused by HEXA-B gene. HEXA deficiency is seen in Tay sachs and HEXB defic...
Detailed Description
This study is a case- control, open label clinical trial. Patients are all registered with diagnosis of Sandhoff and Tay sachs, and recruited at children's medical center Tehran-IRAN. Diagnosis is con...
Eligibility Criteria
Inclusion
- Clinically and enzymatically suspected infants of Sandhoff (SD)/Tay-Sachs (TSD) diseases followed confirmation by molecular study.
Exclusion
- Renal impairment
- Loss of follow up
- Other systemic diseases
- Concomitant drug therapy which may affect neurological system function
Key Trial Info
Start Date :
January 14 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 10 2025
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT03822013
Start Date
January 14 2019
End Date
September 10 2025
Last Update
September 17 2025
Active Locations (3)
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1
Kashan University Of Medical Sciences
Kashan, Isfahan, Iran
2
Mashhad University Of Medical Sciences
Mashhad, Khorasan, Iran
3
Tehran University Of Medical Sciences
Tehran, Tehran Province, Iran