Stem cell trial aims to heal wounds in 'Butterfly Skin' disease
NCT ID NCT05838092
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
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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 Hide 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
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Masonic Cancer Center and Medical Center Minneapolis
Minneapolis, Minnesota, 55455, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Gene therapy for 'Butterfly Skin' disease tracked for 15 years in major safety registry
- Anti-Inflammatory drug may ease swallowing in rare skin disease
- New eye drop battle: which offers better dry eye relief?
- New hope for blistering skin diseases: drug targets antibodies to heal wounds
- Stanford launches study to better understand rare blistering skin disease
- Gene therapy gel aims to stop skin cancer from coming back in RDEB patients