Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III)

• ClinicalTrials.gov Identifier: NCT05838092
• Recruitment Status: Recruiting
• First Posted: May 1, 2023
• Last Update Posted: October 30, 2023
• Sponsor: RHEACELL GmbH & Co. KG
• Information provided by (Responsible Party): RHEACELL GmbH & Co. KG

Tracking Information

• First Submitted Date: April 20, 2023
• First Posted Date: May 1, 2023
• Last Update Posted Date: October 30, 2023
• Actual Study Start Date: October 18, 2023
• Estimated Primary Completion Date: December 1, 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 20, 2023)

Proportion of subjects with complete target wound closure [ Time Frame: 12 months ]

Proportion of subjects with complete healing of target wound, determined by the investigator, as compared to baseline

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures (submitted: April 20, 2023): Time to complete target wound closure from baseline [ Time Frame: 12 months ]
• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III)
• 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)
• Brief 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Epidermolysis Bullosa

Intervention

• Drug: allo-APZ2-OTS
  Allogeneic dermal ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs)
• Drug: Placebo
  Placebo

Study Arms

• Experimental: Verum
  Intervention: Drug: allo-APZ2-OTS
• Placebo Comparator: Placebo
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: April 20, 2023): 74
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 1, 2025
• Estimated Primary Completion Date: December 1, 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 6 Months and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Cristina Daniele; +49 6221 71833-66; cristina.daniele@rheacell.com

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT05838092
• Other Study ID Numbers: allo-APZ2-EB-III-01
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: RHEACELL GmbH & Co. KG
• Original Responsible Party: Same as current
• Current Study Sponsor: RHEACELL GmbH & Co. KG
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: RHEACELL GmbH & Co. KG
• Verification Date: October 2023