Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB

Related Clinical Trial
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

Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB

Official Title

Single Center, Single Group Assignment, Open Label Trial to Assess Safety and Effectiveness of Intravenous Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With Recessive Dystrophic Epidermolysis Bullosa

Brief Summary

      Previously, many studies have been conducted on mesenchymal stem cells derived from bone
      marrow or subcutaneous fat, but interest in cord blood-derived mesenchymal stem cell
      treatments has been increasing recently.

      In the case of cord blood as a source, the isolation of mesenchymal stem cells is easier than
      bone marrow or fat tissue, and cord blood-derived mesenchymal stem cells have an advantage as
      a treatment because they have faster population doubling time.

      To date, no clinical research on the treatment of patients using cord blood-derived
      mesenchymal stem cells has been reported in the literature, but there have already been
      registered at clinicaltrials.gov and currently being conducted overseas.

      In this study, we will study the safety and effectiveness of RDEB patient treatment using
      cord blood-derived mesenchymal stem cells with these advantages.
    

Detailed Description

      Until now, all clinical trials for Recessive Dystrophic Epidermolysis Bullosa (RDEB) have
      examined the potential of bone marrow-derived MSCs. However, umbilical cord blood (UCB) is
      another important source of stem cells, since its non-invasive collection procedure and rapid
      availability from cord blood banking. Human UCB-derived MSCs (hUCB-MSCs) exhibit high
      proliferation capacity and low immunogenicity. A few data support that UCB-MSCs may have
      significantly greater immunosuppressive potential than other sources of MSCs. A preclinical
      study has demonstrated that systemic infusions of human UCB-derived unrestricted somatic stem
      cells, a subpopulation of non-hematopoietic stromal stem cells, significantly extended the
      life span and reduced blistering of RDEB mice model. Given the promising results of the
      preclinical study, we conducted a first-in-human, phase 1/2a clinical trial of intravenous
      administrations of allogeneic hUCB-MSCs in patients with RDEB to determine the safety,
      tolerability, and potential efficacy.
    

Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Adverse events related to the intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell

Secondary Outcome

 Change in type VII collagen and anchoring fibril expression at dermoepidermal junction

Condition

Recessive Dystrophic Epidermolysis Bullosa

Intervention

Human Umbilical Cord Blood-derived Mesenchymal Stem Cells

Study Arms / Comparison Groups

 FURESTEM-CD Inj
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

5

Start Date

October 13, 2016

Completion Date

January 10, 2020

Primary Completion Date

January 10, 2020

Eligibility Criteria

        Inclusion Criteria:

          1. Patients who diagnosed with recessive dystrophic epidermolysis bullosa through
             clinical, histological(Partial or complete loss of VII collagen (C7) should be
             confirmed by DIF and electron microscopy examination) and genetic testing(COL7A1
             Genetic mutation must be confirmed).

          2. RDEB patients aged 10 to 60 years old (In the case of patients under the age of 19,
             patients who obtain consent from a representative (parental authority or guardian))

          3. Patients who have heard the purpose and contents of a clinical trial and voluntarily
             signed the consent form prior to the clinical trial (Legal representative in case of
             minor)

          4. Patients who can be monitored during a clinical trial period

        Exclusion Criteria:

          1. Patients who disagree with this study

          2. Patients who is not accompanied by a guardian if those with impaired consent ability

          3. Patient or the patient's representative is unable to hear and understand the
             explanation

          4. In case of received immunotherapy or chemotherapy including oral corticosteroid
             (topical treatment is possible) for more than 1 week within 8 weeks before
             registration.

          5. All kinds of live vaccines except influenza vaccine within four weeks prior to
             registration

          6. Clinically significant infections within four weeks of the screening date or during
             the screening period (pneumonia, pyelonephritis, Clostridium difficile etc)

          7. All kinds of confirmed congenital or acquired immunodeficiency syndrome

          8. Acute, chronic infection (Type B, Type C) corresponding to:

             - HBs-Ag, IgM anti-HBc, IgG anti-HBc positive (However, if HBs-Ag and IgM anti-HBc is
             negative, but only IgG anti-HBc is positive, if ani-HBs Ab positive, this clinical
             trial can be registered.)

          9. Patients who with allogenic stem cell treatment experience within 1 year from the
             screening test date

         10. Patients who have a history of malignant tumors or is currently being treated
             (squamous cell carcinoma of the skin, cutaneous squamous cell carcinoma inclusion)

         11. Type VII collagen ELISA positive and IIF positive

         12. Pregnant or lactating women (Women of childbearing potential should agree to use
             appropriate contraceptive methods (hormonal or barrier method of contraception or
             abstinence) prior to enrollment in the study and during the study period, including
             one month after the last administration of the test drug. If pregnant or suspected of
             being pregnant while participating in the study, the investigator should be informed
             immediately.)

         13. Other cases where the researcher judges that participation in this clinical trial is
             inappropriate

         14. If other clinical trial drugs have been administered within 4 weeks prior to
             registration or are currently participating in a clinical trial
      

Gender

All

Ages

10 Years - 60 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Korea, Republic of

Location Countries

Korea, Republic of

Administrative Informations


NCT ID

NCT04520022

Organization ID

3-2015-0285


Responsible Party

Principal Investigator

Study Sponsor

Gangnam Severance Hospital

Collaborators

 Daewoong Pharmaceutical Co. LTD.

Study Sponsor

, , 


Verification Date

August 2020