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Desperate hope: stem cells tested on one child with rare muscle disease

NCT ID NCT05154851

Disease control Sponsor: Hope Biosciences Research Foundation 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 Results may not be published yet.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
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) This study
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This status has not been confirmed recently, so it may be out of date.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 trial gives a single child with a rare form of congenital muscular dystrophy access to their own banked stem cells. The cells are given through 14 IV infusions to see if they are safe and can help control the disease. Because it involves only one patient, the results will be very limited.

What this could mean

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

Active substance
autologous adipose-derived mesenchymal stem cells (HB-adMSCs)
What this could lead to
If successful, this could point toward a way to slow or manage congenital muscular dystrophy in this patient.
What could go wrong
This is a single-patient expanded access study, not a full clinical trial. Results may not apply to others, and risks include infection or infusion reactions.

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.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

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: 1. Subject diagnosed with LCMNA congenital muscular dystrophy. 2. Subject must have banked his stem cells at Hope Biosciences LLC. Exclusion Criteria: 1. Subject has any active infection requiring medications. 2. The subject has any known coagulation anomalies.

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Conditions

The condition(s) this trial relates to.

congenital muscular dystrophy due to LMNA mutation muscular dystrophy

As listed by the trial registrant

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

Congenital muscular dystrophy due to lamin A/C mutation

Contacts and locations

Locations

  • Hope Biosciences Stem Cell Research Foundation

    Sugar Land, Texas, 77478, United States