Miglustat therapy for infants with Sandhoff and Tay-Sachs diseases
Survey of Miglustat Therapeutic Effects on Neurological and Systemic Symptoms of Infantile Type of Sandhoff and Taysachs Diseases
This study is testing whether Miglustat therapy can help improve the health and quality of life for infants with Sandhoff and Tay-Sachs diseases.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 6 Months to 24 Months |
| Sex | All |
| Sponsor | Tehran University of Medical Sciences Academic / other |
| Locations | 3 sites (Kashan, Isfahan and 2 other locations) |
| Trial ID | NCT03822013 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the effects of Miglustat therapy on infants diagnosed with Sandhoff and Tay-Sachs diseases, both of which are severe genetic disorders. The study is designed as a case-control, open-label trial where one group of patients receives Miglustat for one year while a control group does not. Patients are assessed regularly for neurological function, seizure activity, and quality of life using validated measures. The trial aims to gather data on the efficacy of Miglustat in improving clinical outcomes for these patients.
Who should consider this trial
Good fit: Ideal candidates for this study are infants clinically and enzymatically diagnosed with Sandhoff or Tay-Sachs diseases.
Not a fit: Patients with renal impairment, other systemic diseases, or those on concomitant drug therapy affecting neurological function may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could significantly improve neurological function and quality of life for infants suffering from these devastating conditions.
How similar studies have performed: While Miglustat has been previously used for other lysosomal storage diseases, this specific application for Sandhoff and Tay-Sachs diseases is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Kashan, Isfahan and 2 other locations
- Kashan University Of Medical Sciences — Kashan, Isfahan, Iran, Islamic Republic of (Recruiting)
- Mashhad University Of Medical Sciences — Mashhad, Khorasan, Iran, Islamic Republic of (Recruiting)
- Tehran University Of Medical Sciences — Tehran, Iran, Islamic Republic of (Recruiting)
Study contacts
- Study coordinator: Alireza Tavasoli, MD
- Email: alirezatavasoli236@gmail.com
- Phone: 00989155130257
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.