Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)
Launched by TEHRAN UNIVERSITY OF MEDICAL SCIENCES · Jan 29, 2019
Trial Information
Current as of June 26, 2025
Recruiting
Keywords
ClinConnect Summary
The clinical trial titled "Effects of Miglustat Therapy on Infantile Type of Sandhoff and Tay Sachs Diseases" is investigating a potential treatment for two serious genetic conditions known as Sandhoff disease and Tay-Sachs disease. These conditions affect the body's ability to break down certain fats, leading to harmful buildup in the brain and nervous system. Currently, treatment focuses on supportive care, like managing nutrition and seizures, but researchers are exploring whether the drug Miglustat can improve symptoms related to these diseases. Miglustat has previously shown promise in other similar conditions by helping to reduce some neurological issues, although results have varied.
To participate in this trial, infants diagnosed with Sandhoff or Tay-Sachs diseases may qualify if they meet specific criteria, such as being confirmed through genetic testing. However, individuals with kidney problems or other serious health issues may not be eligible. Participants will receive Miglustat and will be monitored for its effects, including any side effects like weight loss or digestive issues. The hope is that this research will help find an effective treatment that can improve the quality of life for affected children and their families.
Gender
ALL
Eligibility 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
About Tehran University Of Medical Sciences
Tehran University of Medical Sciences (TUMS) is a prestigious institution dedicated to advancing healthcare through innovative research and education. As a leading clinical trial sponsor, TUMS is committed to conducting high-quality, ethically sound clinical research that contributes to the understanding and treatment of various medical conditions. With a strong emphasis on collaboration and interdisciplinary approaches, TUMS harnesses the expertise of its diverse faculty and state-of-the-art facilities to facilitate groundbreaking studies that aim to improve patient outcomes and enhance public health. Through its rigorous oversight and adherence to regulatory standards, TUMS ensures the integrity and reliability of its clinical trials, fostering trust and transparency in the research community.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Tehran, , Iran, Islamic Republic Of
Mashhad, Khorasan, Iran, Islamic Republic Of
Kashan, Isfahan, Iran, Islamic Republic Of
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
Similar Trials