- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05464381
Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over)
February 22, 2023 updated by: RHEACELL GmbH & Co. KG
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)
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.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Anticipated)
74
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Cristina Daniele
- Phone Number: T +49 6221 71833-66
- Email: cristina.daniele@rheacell.com
Study Locations
-
-
-
Salzburg, Austria, 5020
- Recruiting
- EB-Haus Austria; Salzburger Landeskliniken (SALK)
-
-
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.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Male or female subjects with a diagnosis of RDEB or JEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping (IFM);
- Subject is eligible to participate in this clinical trial based on general health condition;
- Subject with a target wound meeting the following criteria: 5-50 cm2, ≥ 21 days and < 9 months, no signs of acute infection;
- Patient/legal representative understands the nature of the procedure and provide written informed consent/assent prior to any clinical trial procedure;
- 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:
- Any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma;
- Any known allergies to components of the IP or premedication;
- Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
- Pregnant or lactating women;
- Current or previous (within 30 days of screening) treatment with another IP, or participation and/or under follow-up in another interventional clinical trial;
- Previous participation in this clinical trial (except for screening failures);
- 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
- Employees of the sponsor, or employees or relatives of the investigator.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: Placebo
|
Placebo
|
Experimental: Verum
|
Allogeneic dermal ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs)
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Proportion of subjects with complete target wound closure
Time Frame: 6 months
|
Proportion of subjects with complete healing of target wound, determined by the investigator, as compared to baseline
|
6 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
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 Major Dates
Study Start (Actual)
February 1, 2023
Primary Completion (Anticipated)
December 1, 2024
Study Completion (Anticipated)
December 1, 2025
Study Registration Dates
First Submitted
July 5, 2022
First Submitted That Met QC Criteria
July 18, 2022
First Posted (Actual)
July 19, 2022
Study Record Updates
Last Update Posted (Actual)
February 24, 2023
Last Update Submitted That Met QC Criteria
February 22, 2023
Last Verified
February 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- allo-APZ2-EB-III
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
UNDECIDED
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Epidermolysis Bullosa
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Castle Creek Pharmaceuticals, LLCCompletedDystrophic Epidermolysis Bullosa | Epidermolysis Bullosa Simplex | Junctional Epidermolysis Bullosa | Epidermolysis Bullosa (EB)United States
-
Krystal Biotech, Inc.CompletedDystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Dominant Dystrophic Epidermolysis Bullosa | DEB - Dystrophic Epidermolysis BullosaUnited States
-
Krystal Biotech, Inc.CompletedDystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Dominant Dystrophic Epidermolysis BullosaUnited States
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Castle Creek Biosciences, LLC.TerminatedEpidermolysis Bullosa Dystrophica, RecessiveUnited States
-
Instituto de Investigación Hospital Universitario...Instituto de Salud Carlos III; Universidad Carlos III Madrid (TERMeG); St John... and other collaboratorsUnknownEpidermolysis Bullosa Dystrophica, RecessiveSpain
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Lenus Therapeutics, LLCTerminatedDystrophic Epidermolysis Bullosa | Junctional Epidermolysis BullosaUnited States
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Phoenicis TherapeuticsNot yet recruitingDystrophic Epidermolysis BullosaUnited States
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Thomas Jefferson UniversityOnconova Therapeutics, Inc.RecruitingRecessive Dystrophic Epidermolysis BullosaUnited States
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