Phase III clinical trial of ABCB5+ MSCs for the treatment of EB
- Conditions
- Recessive Dystrophic Epidermolysis Bullosa(RDEB) and Junctional Epidermolysis Bullosa (JEB)MedDRA version: 20.0Level: PTClassification code: 10014989Term: Epidermolysis bullosa Class: 100000004850Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2022-500266-10-00
- Lead Sponsor
- Rheacell GmbH & Co. KG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 163
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;
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method