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Phase III clinical trial of ABCB5+ MSCs for the treatment of EB
EU CTIS ID: 2022-500266-10-00
What this study is testing
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.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Male or female subjects from birth 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;
- 2. Subject is eligible to participate in this clinical trial based on general health condition assessed by the following specific lab values: a. Hematology: Absolute neutrophil count > 1000/mm3 and platelet count > 150,000/mcL; b. Hepatic: AST and ALT < 2x the upper limit of normal for age; c. Renal: Creatinine < 2x the upper limit of normal for age; d. Pulmonary: Oxygen saturation > 92% on room air and without supplemental oxygen requirement;
- 3. Subject with a target wound meeting the following criteria: 5-50 cm2, 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.
You likely can't join if
- 1. 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;
- 10. Previous participation in this clinical trial** (except for screening failures);
- 11. 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;
- 12. Employees of the sponsor, or employees or relatives of the investigator.
- *IP: Ringer lactate, glucose, DMSO ** Except for subjects enrolled under protocols issued before 2024. Re-screening may only be performed after a thorough benefit/risk assessment performed by the investigator.
- 2. Known chronic lung disease;
See the full eligibility criteria
- 1. Male or female subjects from birth 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;
- 2. Subject is eligible to participate in this clinical trial based on general health condition assessed by the following specific lab values: a. Hematology: Absolute neutrophil count > 1000/mm3 and platelet count > 150,000/mcL; b. Hepatic: AST and ALT < 2x the upper limit of normal for age; c. Renal: Creatinine < 2x the upper limit of normal for age; d. Pulmonary: Oxygen saturation > 92% on room air and without supplemental oxygen requirement;
- 3. Subject with a target wound meeting the following criteria: 5-50 cm2, 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.
- 1. 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;
- 10. Previous participation in this clinical trial** (except for screening failures);
- 11. 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;
- 12. Employees of the sponsor, or employees or relatives of the investigator.
- *IP: Ringer lactate, glucose, DMSO ** Except for subjects enrolled under protocols issued before 2024. Re-screening may only be performed after a thorough benefit/risk assessment performed by the investigator.
- 2. Known chronic lung disease;
- 3. Clinically significant laboratory values for coagulation and thrombocytes at screening;
- 4. Thromboembolic events of any grade in medical history;
- 5. Immunoreactions of any grade in medical history;
- 6. Any known allergies to components of the IP* or premedication;
- 7. Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
- 8. Pregnant or lactating women;
- 9. Current or previous (within 30 days of screening) treatment with another IP, or participation and/or under follow-up in another interventional clinical trial;
The study team makes the final eligibility decision.
Where it's taking place
- Israel
- Chile
- United Kingdom
- Argentina
- Taiwan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Israel; Chile; United Kingdom; Argentina; Taiwan. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.