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

Related Clinical Trial
Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over) Growth Hormone in EB Dose-ranging Study of Dentoxol® Mouthrinse for Managing Oral Symptoms in People With Epidermolysis Bullosa. Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa Extension Study to PTR-01-002 Long-Term Follow-up Protocol A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa Improve Adherence to Weak or Strong Opioid Analgesics at the Time of Care in Children With Hereditary Epidermolysis Bullosa Gynecological Follow-up of Patients With Dystrophic Epidermolysis Bullosa (EBD) Topical Gentamicin Nonsense Suppression Therapy of EB The Efficacy and Safety of 3% Cannabidiol (CBD) Cream in Patients With Epidermolysis Bullosa: A Phase II/III Trial A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB The Objective of This Study is to Compare the Efficacy and Safety of Beremagene Geperpavec (B-VEC) Topical Gel With That of Placebo for the Treatment of Dystrophic Epidermolysis Bullosa (DEB). Trial To Assess Efficacy Of A Chimeric Skin In Patients With Epidermolysys Bullosa Randomised Double Blind Placebo Controlled Cross Over Design of the Efficacy of Topical Morphine for Inflammatory Pain in Children With Epidermolysis Bullosa Study of Cellutome System for Treatment of Individual Lesions in EB Pts Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa Biochemical Correction of Severe EB by Allo HSCT and “Off-the-shelf” MSCs Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa Study of the Nutritional, Metabolic, and Body Composition Profile in Children and Adolescents With Epidermolysis Bullosa MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs Randomised Double Blind Crossover Placebo Controlled Study to Evaluate the Efficacy of Tetracycline in Epidermolysis Bullosa Observational Study of a Cohort of Patients With Hereditary Epidermolysis Bullosa Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa A Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis Bullosa Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa Evaluation of the Efficacy of ROPIVACAINE in Children and Young Adults With Hereditary Epidermolysis Bullosa Oleogel-S10 in Wound Healing of Inherited Epidermolysis Bullosa (BEB-10) The Efficacy of Trimethoprim in Wound Healing of Patients With Epidermolysis Bullosa A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa A Follow-up Study to Evaluate the Efficacy and Safety of ALLO-ASC-DFU in ALLO-ASC-EB-101 Clinical Trial Proof of Concept Study for a Dressing Glove Pregabalin Treatment for RDEB Pain and Itch Study of Alwextin® Cream in Treating Epidermolysis Bullosa A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing Uses of Irradiated Human Amniotic Membrane in the Treatment of Dystrophic Epidermolysis Bullosa Patients Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa A Comparative Study of the Healing of Chronic Ulcers of Recessive Epidermolysis Bullosa : Dressing vs Amniotic Membrane Topical QR-313 in Dominant Dystrophic Epidermolysis Bullosa (DDEB) or Recessive Dystrophic Epidermolysis Bullosa (RDEB) Due to Mutation(s) in Exon 73 of the COL7A1gene Rigosertib for RDEB-SCC Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa Short Term Observational Study in DEB Patients Gentamicin for RDEB Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB. Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients Self-Assembled Skin Substitute for the Treatment of Epidermolysis Bullosa Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa Topical Beremagene Geperpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB) A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Molecular Signatures of Cutaneous Squamous Cell Carcinoma During Recessive Dystrophic Epidermolysis Bullosa Treatment of Chronic and Non-Chronic Wounds in Patients With Recessive Dystrophic Epidermolysis Bullosa Using Helicoll Collagen Dressings Versus Standard of Care Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) MSC EVs in Dystrophic Epidermolysis Bullosa Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa Prospective, Longitudinal Natural History Study in Dystrophic Epidermolysis Bullosa Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer Study of Immune Tolerance and Capacity for Wound Healing of Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate) Characteristics of Adult Patients With Recessive Dystrophic Epidermolysis Bullosa Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa Characteristics of Patients With Recessive Dystrophic Epidermolysis Bullosa Study to Evaluate the Safety of ALLO-ASC-DFU in the Subjects With Dystrophic Epidermolysis Bullosa The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB) A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa

Brief Title

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

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)

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.
    


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Proportion of subjects with complete target wound closure


Condition

Epidermolysis Bullosa

Intervention

allo-APZ2-OTS

Study Arms / Comparison Groups

 Verum
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

74

Start Date

December 2022

Completion Date

December 2025

Primary Completion Date

December 2024

Eligibility Criteria

        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.
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

, , 



Administrative Informations


NCT ID

NCT05464381

Organization ID

allo-APZ2-EB-III


Responsible Party

Sponsor

Study Sponsor

RHEACELL GmbH & Co. KG


Study Sponsor

, , 


Verification Date

July 2022