ACTIVE_NOT_RECRUITING

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

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)

Quick Facts

Study Start:2023-10-18

Study Completion:2026-07

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05838092

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Months

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	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.

Inclusion Criteria

Exclusion 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.

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

Study Locations (Sites)

Children's Hospital Colorado

Aurora, Colorado, 80045

United States

Masonic Cancer Center and Medical Center Minneapolis

Minneapolis, Minnesota, 55455

United States

Collaborators and Investigators

Sponsor: RHEACELL GmbH & Co. KG

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-10-18

Study Completion Date2026-07

Study Record Updates

Study Start Date2023-10-18

Study Completion Date2026-07

Terms related to this study

Additional Relevant MeSH Terms

Epidermolysis Bullosa