Stem cell infusion aims to buy time for kids with fatal brain disease

NCT ID NCT06030648

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests whether a single infusion of donated mesenchymal stem cells is safe for children with cerebral adrenoleukodystrophy (cALD), a rare and life-threatening brain disease. The stem cells are given as a temporary bridge to buy time before a more definitive treatment like a bone marrow transplant or gene therapy. Only 10 participants are being enrolled, and the main goal is to check for side effects.

What this could mean

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

Active substance

mesenchymal stem cells (MSC)

What this could lead to

If it works, this could provide a safe way to stabilize brain inflammation in children with cerebral adrenoleukodystrophy while they wait for a stem cell transplant or gene therapy.

What could go wrong

This is a very early Phase 1 trial with only 10 participants, so it is primarily checking safety, not effectiveness. The study is currently suspended, and the treatment is only a temporary bridge, not a cure.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for CEREBRAL ADRENOLEUKODYSTROPHY are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

adrenoleukodystrophy X-linked cerebral adrenoleukodystrophy

As listed by the trial registrant

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

Contacts and locations

Locations

  • Masonic Cancer Center, University of Minnesota

    Minneapolis, Minnesota, 55455, United States