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

Sponsor
RHEACELL GmbH & Co. KG (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05464381
Collaborator
(none)
74
Enrollment
2
Arms
36
Anticipated Duration (Months)

Study Details

Study Description

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. 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.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
74 participants
Allocation:
Randomized
Intervention Model:
Crossover 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)
Anticipated Study Start Date :
Dec 1, 2022
Anticipated Primary Completion Date :
Dec 1, 2024
Anticipated Study Completion Date :
Dec 1, 2025

Arms and Interventions

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

Primary Outcome Measures

  1. Proportion of subjects with complete target wound closure [6 months]

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

Eligibility Criteria

Criteria

Ages Eligible for Study:
N/A and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:

  1. Male or female subjects with a diagnosis of RDEB or JEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping (IFM);

  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

Locations

No locations specified.

Sponsors and Collaborators

  • RHEACELL GmbH & Co. KG

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
RHEACELL GmbH & Co. KG
ClinicalTrials.gov Identifier:
NCT05464381
Other Study ID Numbers:
  • allo-APZ2-EB-III
First Posted:
Jul 19, 2022
Last Update Posted:
Jul 19, 2022
Last Verified:
Jul 1, 2022
Individual Participant Data (IPD) Sharing Statement:
Undecided
Plan to Share IPD:
Undecided
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Epidermolysis Bullosa

Study Results

No Results Posted as of Jul 19, 2022