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

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

Study Description
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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.

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Drug: allo-APZ2-OTS Drug: Placebo Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 74 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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)
Actual Study Start Date : October 18, 2023
Estimated Primary Completion Date : December 1, 2024
Estimated Study Completion Date : December 1, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Verum Drug: allo-APZ2-OTS
Allogeneic dermal ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs)
Placebo Comparator: Placebo Drug: Placebo
Placebo


Outcome Measures
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Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Time to complete target wound closure from baseline [ Time Frame: 12 months ]

Eligibility Criteria
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Ages Eligible for Study:   6 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05838092


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

Locations
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United States, Minnesota
Masonic Cancer Center and Medical Center Minneapolis Recruiting
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
RHEACELL GmbH & Co. KG
More Information
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Responsible Party: RHEACELL GmbH & Co. KG
ClinicalTrials.gov Identifier: NCT05838092    
Other Study ID Numbers: allo-APZ2-EB-III-01
First Posted: May 1, 2023    Key Record Dates
Last Update Posted: October 30, 2023
Last Verified: October 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
 Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous