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Stem cell trial aims to heal wounds in 'Butterfly Skin' disease

NCT ID NCT05838092

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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 trial tests whether ABCB5-positive stem cells given through an IV can safely and effectively heal wounds in people with epidermolysis bullosa, a rare genetic condition that causes fragile, blistering skin. The study includes 9 participants with a specific type of the disease (recessive dystrophic EB) and compares the stem cells to a placebo. The main goal is to see if the target wound closes completely.

What this could mean

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

Active substance
ABCB5-positive mesenchymal stromal cells (stem cells)
What this could lead to
If it works, this could point toward a treatment that helps heal wounds in people with epidermolysis bullosa, a severe skin condition.
What could go wrong
This is a very small early-stage trial with only 9 participants, so results may not apply to everyone. The treatment may not work better than placebo, and there could be side effects from the stem cells.

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

9 people

The number who actually took part.

Started

Oct 2023

Expected to finish

Jul 2026

An estimate. End dates often move.

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

6 months 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: 1. Male or female subjects from 6 months of age with a diagnosis of RDEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping 2. Subject is eligible to participate in this clinical trial based on general health condition; 3. Subject with a target wound meeting the following criteria: 5-50 cm2, ≥ 21 days and \< 9 months, no signs of acute infection; 4. Patient/legal representative understands the nature of the procedure and provide written informed consent/assent prior to any clinical trial procedure; 5. Women of childbearing potential must have a negative urine pregnancy test at Visit 1. Women of childbearing potential, male participants, and their partner must be willing to use highly effective contraceptive methods during the course of the entire clinical trial. Exclusion Criteria: 1. Any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma; 2. Any known allergies to components of the IP or premedication; 3. Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol; 4. Pregnant or lactating women; 5. Current or previous (within 30 days of screening) treatment with another IP, or participation and/or under follow-up in another interventional clinical trial; 6. Previous participation in this clinical trial (except for screening failures); 7. Clinically significant or unstable concurrent disease or other clinical contraindications like an uncontrolled or poorly controlled mental health condition of the subject and/or his/her legal representative that could impact on patient's safety or interfere with study compliance such as inability to attend scheduled study visits; Confidential 8. Employees of the sponsor, or employees or relatives of the investigator.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Masonic Cancer Center and Medical Center Minneapolis

    Minneapolis, Minnesota, 55455, United States

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