Experimental drug zavesca tested for rare fatal brain diseases in infants

NCT ID NCT03822013

Terminated Disease control Sponsor: Tehran University of Medical Sciences Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This phase 3 trial tested the drug miglustat (Zavesca) in 30 infants with Sandhoff or Tay-Sachs diseases, rare genetic disorders that destroy nerve cells. The goal was to see if the drug could reduce hospitalizations, seizures, and feeding problems while improving motor function. The study was terminated early, but results may still help guide future research.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Miglustat (Zavesca)
What this could lead to: If it works, this could point toward a treatment that slows neurological decline and improves quality of life for infants with these rare, fatal diseases.
What could go wrong: The trial was terminated early with only 30 participants, so results are limited. Previous studies have shown mixed benefits, and side effects like weight loss and diarrhea are common.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

GM2 gangliosidosis Sandhoff disease Tay-Sachs disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GM2 GANGLIOSIDOSIS SUPPORTIVE CARE

Contacts and locations

Locations

Kashan University Of Medical Sciences

Kashan, Isfahan, Iran
Mashhad University Of Medical Sciences

Mashhad, Khorasan, Iran
Tehran University Of Medical Sciences

Tehran, Tehran Province, Iran

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