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

NCT ID NCT06030648

Disease control Sponsor: Masonic Cancer Center, University of Minnesota 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 This study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 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.

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 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 10 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2023

Expected to finish

May 2029

An estimate. End dates often move.

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

3 years and older

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: * age ≥ 3 years * diagnosis of ALD, as established by elevation of very long chain fatty acid levels or gene mutation * evidence of active cerebral disease as determined by the presence of gadolinium enhancement * ALD MRI (Loes) score ≥ 1 * Patients who have not received prior gene therapy or transplant * Life expectancy of \> 6 months as determined by the enrolling researcher * Have adequate organ function confirmed by laboratory values obtained within 28 days prior to enrollment Exclusion Criteria: * Inability to undergo sedation or MRI studies for any reason * Other concurrent life-threatening disease (life expectancy \<6 months) or eligible for hospice care * Known or suspected hypersensitivity to human serum albumin, diphenhydramine, acetaminophen, methylprednisolone, or any of the components of IMS001.

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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.

Cerebral adrenoleukodystrophy

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Masonic Cancer Center, University of Minnesota

    Minneapolis, Minnesota, 55455, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.