Last Verified: | February/29/2020 |
First Submitted: | January/21/2019 |
Estimated Enrollment Submitted: | January/28/2019 |
First Posted: | January/29/2019 |
Last Update Submitted: | March/29/2020 |
Last Update Posted: | March/30/2020 |
Actual Study Start Date: | January/13/2019 |
Estimated Primary Completion Date: | August/31/2021 |
Estimated Study Completion Date: | December/29/2021 |
Study Type: | Interventional |
Allocation: | Randomized |
: | Although randomized control trial is the gold standard for clinical trial studies; there are ethical concerns about placebo control group in rare diseases such as Sandhoff and Tay sachs diseases. |
Primary Purpose: | Supportive Care |
Masking: | None (Open Label) |
Arm | Intervention/treatment |
---|---|
Experimental: Miglustat Miglustat is administered, dose is adjusted according to Body Surface Area as below: >1.25 : 200 mg TDS 0.88-1.25 : 200mg BID 0.73-0.88 :100mg TDS 0.47-0.73 : 100mg BID <0.47 :100mg daily | Drug: Miglustat Treatment with Zavesca regimen based on body surface area as follows: SQRT [Height (cm) × Weight (kg)] / 3600 <1.25 : 200mg TDS 0.88- 1.25: 200mg BID 0.73- 0.88: 100 mg TDS 0.47- 0.73: 100 mg BID <0.47: 100 mg Daily |
No Intervention: No Miglustat |
Ages Eligible for Study: | 6 Months to 6 Months |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | Yes |
Criteria: | Inclusion Criteria: - Clinically and enzymatically suspected infants of Sandhoff (SD)/Tay-Sachs (TSD) diseases followed confirmation by molecular study. Exclusion Criteria: - Renal impairment - Loss of follow up - Other systemic diseases - Concomitant drug therapy which may affect neurological system function |