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Experimental drug zavesca tested for rare fatal brain diseases in infants

NCT ID NCT03822013

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

30 people

The number who actually took part.

Started

Jan 2019

Finished

Sep 2025

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 to 24 months

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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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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