Epidermolysis BullosaPhase 3NCT05464381
Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over)
A Phase 3 study testing allo-APZ2-OTS in people with Epidermolysis Bullosa, run by RHEACELL GmbH & Co. KG at 16 sites in 12 countries.
Trial summary
The aim of this clinical trial is to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with recessive dystrophic epidermolysis bullosa (RDEB) compared to placebo. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- allo-APZ2-OTS
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 91 participants
- Where
- 16 sitesin 12 countries
- Study dates
- 13 Jun 2023to Jan 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female subjects with a diagnosis of RDEB or JEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping (IFM). A minimal body weight of at least 5 kg is required;
- Subject is eligible to participate in this clinical trial based on general health condition;
- Subject with a target wound meeting the following criteria: 5-50 cm2, and < 9 months, no signs of acute infection;
- Patient/legal representative understands the nature of the procedure and provide written informed consent/assent prior to any clinical trial procedure;
- 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.
Who cannot join
- Signs and/or a history of skin precancerous and cancerous lesions at screening or any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma;
- Known chronic lung disease;
- Clinically significant laboratory values for coagulation and thrombocytes at screening;
- Thromboembolic events of any grade in medical history;
- Immunoreactions of any grade in medical history;
- Any known allergies to components of the IP or premedication;
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- Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
- Pregnant or lactating women;
- Current or previous (within 30 days of screening) treatment with another IP, or participation and/or under follow-up in another interventional clinical trial;
- Previous participation in this clinical trial (except for screening failures);
- 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;
- Employees of the sponsor, or employees or relatives of the investigator.
Trial locations and contacts
16 sites in 12 countries.
16 sites in 12 countries
France3 sites
Universitaetsklinik fuer Dermatologie und Allergologie
Nice
Hospital Necker-Enfants Malades
Paris
Service de Dermatologie Hospital Saint-Louis
Paris
Greece2 sites
Andreas Syggros Hospital of Cutaneous Venereal Diseases
Athens
Venereal & Skin Diseases Hospital of Thessaloniki
Thessaloniki
Italy2 sites
UOSD Pediatria-Alta Intensita di Curà, Ospedale Maggiore
Milan
Ospedale Pediatrico Bambin Gesu
Roma
Austria1 site
EB-Haus Austria; Salzburger Landeskliniken (SALK)
Salzburg, 5020
About this study
Official title: A Double-blind, Randomized, Placebo-controlled, Interventional, Multicenter, Phase III Clinical Trial to Investigate the Safety and Efficacy of ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs) on Epidermolysis Bullosa (EB)
The aim of this clinical trial is to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with recessive dystrophic epidermolysis bullosa (RDEB) compared to placebo. An additional baseline-controlled open-label arm will be included to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with JEB and to RDEB subjects < 1 year.
- Conditions
- Epidermolysis Bullosa
- Being tested
- allo-APZ2-OTS, Placebo
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
allo-APZ2-OTSDrug
Allogeneic dermal ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs)
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Verum | Experimental | Not described Receives: Drug: allo-APZ2-OTS |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects with complete target wound closure
Proportion of subjects with complete healing of target wound, determined by the investigator, as compared to baseline
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2022
- Study start13 Jun 2023
- Primary completion29 Aug 2025
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 91 participants (actual)
Sponsor and investigators
- Lead sponsor
- RHEACELL GmbH & Co. KG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05464381
- Sponsor's protocol ID
- allo-APZ2-EB-III
- Status last verified
- Dec 2025
- First submitted
- 5 Jul 2022
- Last updated
- 10 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-blind, Randomized, Placebo-controlled, Interventional, Multicenter, Phase III Clinical Trial to Investigate the Safety and Efficacy of ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs) on Epidermolysis Bullosa (EB)
- ClinicalTrials.gov ID
- NCT05464381
- Acronym / sponsor ID
- allo-APZ2-EB-III
- Conditions
- Epidermolysis Bullosa
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 91 participants (actual)
- Lead sponsor
- RHEACELL GmbH & Co. KGIndustry (pharma, biotech, devices)
- Study start
- 13 Jun 2023 (actual)
- Primary completion
- 29 Aug 2025 (actual)
- Study completion
- Jan 2027 (estimated)
- First posted
- 19 Jul 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this clinical trial is to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with recessive dystrophic epidermolysis bullosa (RDEB) compared to placebo. An additional baseline-controlled open-label arm will be included to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with JEB and to RDEB subjects < 1 year.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Verum | Experimental | Drug: allo-APZ2-OTS |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female subjects with a diagnosis of RDEB or JEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping (IFM). A minimal body weight of at least 5 kg is required;
- Subject is eligible to participate in this clinical trial based on general health condition;
- Subject with a target wound meeting the following criteria: 5-50 cm2, and < 9 months, no signs of acute infection;
- Patient/legal representative understands the nature of the procedure and provide written informed consent/assent prior to any clinical trial procedure;
- 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.
Key exclusion criteria (12 listed)
- Signs and/or a history of skin precancerous and cancerous lesions at screening or any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma;
- Known chronic lung disease;
- Clinically significant laboratory values for coagulation and thrombocytes at screening;
- Thromboembolic events of any grade in medical history;
- Immunoreactions of any grade in medical history;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| allo-APZ2-OTSAllogeneic dermal ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs) | Drug | Verum |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
Proportion of subjects with complete target wound closure
Time frame: 6 months
Proportion of subjects with complete healing of target wound, determined by the investigator, as compared to baseline
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Dec 2025.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
16 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing allo-APZ2-OTS in people with Epidermolysis Bullosa, run by RHEACELL GmbH & Co. KG at 16 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 10 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in France, Greece, Italy, Argentina, Austria and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of subjects with complete target wound closure (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Dec 2025.