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Can an immune drug slow a rare childhood brain disease?

NCT ID NCT07820579

Symptom relief Sponsor: Sara Elva Espinosa Padilla, MD Source: ClinicalTrials.gov ↗

What the study statuses mean

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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 This study
The trial has finished. Results may not be published yet.
Stopped early
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This status has not been confirmed recently, so it may be out of date.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 Sep 15, 2026 · Last updated Sep 16, 2026 · Updated 1 time

Summary

Ataxia-telangiectasia is a rare, inherited disease that damages the nervous system, skin, and immune system, and it has no cure. Researchers are testing sirolimus, an immune-suppressing drug, in children under 17 with the condition. The six participants take sirolimus by mouth for six months while doctors track side effects, blood test results, and changes in neurological function using a standard ataxia rating scale. The goal is to see whether the drug is safe and whether it might ease symptoms or slow the disease.

What this could mean

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

Active substance
sirolimus, an immune-suppressing drug also known as rapamycin
What this could lead to
If it helps, this could point toward a treatment that slows the nerve damage, skin changes, and immune problems of ataxia-telangiectasia, a disease with no cure.
What could go wrong
This is a very small Phase 2 pilot study of six children, so any benefit seen may not hold up in larger trials. Sirolimus suppresses the immune system and can cause infections, mouth sores, and other side effects.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

6 people

The number who actually took part.

Started

Jul 2024

Finished

Aug 2026

Lead sponsor

A government agency

The lead sponsor is a government body.

Who can take part

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

Ages

Up to 16 years

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: * Patients younger than 17 years with a clinical and genetic diagnosis of ataxia telangiectasia (A-T). * Written informed consent obtained from parents or legal guardians. Exclusion Criteria: * Concomitant oncologic or hematologic diagnosis. * Current treatment with immunosuppressive agents.

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Conditions

The condition(s) this trial relates to.

Ataxia ataxia telangiectasia telangiectasis

As listed by the trial registrant

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

Ataxia-telangiectasia Ataxia-telangiectasia (A-T)

Contacts and locations

Locations

  • Instituto Nacional de Pediatría IRB

    Mexico City, Mexico City, 3700, Mexico

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