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Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa | Clinical Trials | Clareo Health

COMPLETEDPHASE1/PHASE2

Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa

An Interventional, Multicenter, Single Arm, Phase I/IIa Clinical Trial to Investigate the Efficacy and Safety of Allo-APZ2-EB on Epidermolysis Bullosa (EB)

NCT03529877•RHEACELL GmbH & Co. KG

View on ClinicalTrials.gov

Summary

The aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms) and safety (by monitoring adverse events) of three doses of allo-APZ2-EB administered intravenously to patients with recessive dystrophic epidermolysis bullosa (RDEB).

Detailed Description

This is an interventional, single arm, non-randomized, open label, phase I/IIa clinical trial to investigate the efficacy and safety of the IMP allo-APZ2-EB in patients with RDEB. Patients will undergo treatment with the IMP (three repeated intravenous applications) and will be followed up for efficacy for 12 weeks. To assess long-term safety of allo-APZ2-EB one follow-up visit at Month 12 and one follow-up visit at Month 24 post IMP applications is included. Determination of the EB linked symptoms and quality of life will be assessed by using the EBDASI score, the iscorEB, the change in pain and itch perception, and patient's quality of life in EB. The wound healing process will be documented by photography.

Eligibility

Age

0 - 55 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1\. Male or female patients aged between 0 and ≤55 years;
•Staggered design for patient enrollment:
•1. at least 3 adult patients (safety assessment 2 weeks after last treatment of third patient),
•2. at least 3 patients ≥12 to \<18 years (safety assessment 2 weeks after first treatment of third patient),
•3. at least 3 patients ≥5 to \<12 years (safety assessment 2 weeks after first treatment of third patient), and
•4. at least 3 patients ≥12 months to \<5 years;
•5. patients 0 to \<12 months (only in the UK);
•2\. Diagnosed with RDEB (combined diagnosis by genotype assessment \[mutation analysis\] and correlating phenotype assessment \[wound assessment\]), patients must have a negative immunofluorescence test result on salt-split skin against proteins of the basement membrane at Visit 1 (existing test results will be accepted);
•3\. Patient is eligible to participate in this clinical trial based on general health condition at the investigator's discretion;
•US only:
+4 more criteria

Exclusion Criteria

•1. Tumor diseases or history of tumor disease;
•2. Known positive result for human immunodeficiency virus 1 and/or 2;
•3. Any known allergies to components of the IMP;
•4. Evidence of any other medical conditions (such as psychiatric illness or active infection) based on physical examination, or laboratory findings that may interfere with the planned treatment, affect the patient's compliance, or place the patient at high risk of complications related to the treatment; at investigators discretion;
•5. History of prior thrombosis or patients at risk for thrombosis;
•6. Clinically significant or unstable concurrent disease or other clinical contraindications (based upon investigator's judgment);
•7. Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
•8. Pregnant or lactating women;
•9. Current or previous (within 30 days of enrollment) treatment with another IMP, or participation and/or under follow-up in another clinical trial;
•10. Previous participation in this clinical trial (except for screening failures due to an exclusion criterion);
+2 more criteria

Locations (6)

University of Minnesota, Masonic Cancer Center and Medical Center

Minneapolis, Minnesota, United States

EB-Haus Austria; Salzburger Landeskliniken (SALK); Paracelsus Medizinische Privatuniversität Salzburg (PMU)

Salzburg, Austria

Hôpital Saint-Louis; Département de dermatologie

Paris, France

Department of Dermatology, Medical Center-University of Freiburg

Freiburg im Breisgau, Germany

King's College London; St John's Institute of Dermatology;

London, United Kingdom

Great Ormond Street Hospital; Dermatology Department

London, United Kingdom

Key Dates

Start Date

February 16, 2019

Primary Completion Date

November 26, 2021

Completion Date

November 26, 2021

Last Updated

October 7, 2022

Enrollment

ACTUAL participants

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Interventions

allo-APZ2-EB

BIOLOGICAL

A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

NCT04213261

Recessive Dystrophic Epidermolysis Bullosa

View study

ACTIVE_NOT_RECRUITINGPHASE1

Pilot Study of ELK-003 Eye Drops for Treating Ocular Manifestations of Epidermolysis Bullosa

NCT06713434

Junctional Epidermolysis BullosaEpidermolysis Bullosa (EB)+2 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 7, 2022Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa

Inclusion Criteria

Exclusion Criteria

A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

Pilot Study of ELK-003 Eye Drops for Treating Ocular Manifestations of Epidermolysis Bullosa

A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients

Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)

A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients