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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04520022
Recruitment Status : Completed
First Posted : August 20, 2020
Last Update Posted : August 20, 2020
Sponsor:
Collaborator:
Daewoong Pharmaceutical Co. LTD.
Information provided by (Responsible Party):
Sang Eun Lee, Gangnam Severance Hospital

Study Description
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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.


Condition or disease Intervention/treatment Phase
Recessive Dystrophic Epidermolysis Bullosa Drug: Human Umbilical Cord Blood-derived Mesenchymal Stem Cells Phase 1 Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : October 13, 2016
Actual Primary Completion Date : January 10, 2020
Actual Study Completion Date : January 10, 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: FURESTEM-CD Inj Drug: Human Umbilical Cord Blood-derived Mesenchymal Stem Cells
3.0 x 106 cells/kg, IV, Total of 3 doses every 2weeks
Other Names:
  • hUCB-MSCs
  • FURESTEM-CD Inj


Outcome Measures
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Primary Outcome Measures :
  1. Adverse events related to the intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell [ Time Frame: 8 months ]

Secondary Outcome Measures :
  1. Change in type VII collagen and anchoring fibril expression at dermoepidermal junction [ Time Frame: baseline, day 56 ]
  2. Change in Birmingham Epidermolysis Bullosa Severity Score (BEBSS) [ Time Frame: baseline, day56, day 112, day168 ]
  3. Change in Global severity score [ Time Frame: baseline, day56, day 112, day168 ]
  4. Change in total body surface area affected by RDEB [ Time Frame: baseline, day56, day 112, day168 ]
  5. Change in Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire [ Time Frame: baseline, day56, day 112, day168 ]
  6. Change in blister count [ Time Frame: baseline, day56, day 112, day168 ]
  7. Change in pruritus visual analogue scale (VAS) [ Time Frame: baseline, day56, day 112, day168 ]
  8. Change in pain visual analogue scale (VAS) [ Time Frame: baseline, day56, day 112, day168 ]

Eligibility Criteria
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Ages Eligible for Study:   10 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04520022


Locations
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Korea, Republic of
GangnamSeverance Hospital
Seoul, Korea, Republic of
Sponsors and Collaborators
Gangnam Severance Hospital
Daewoong Pharmaceutical Co. LTD.
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Sang Eun Lee, Assistant professor, Gangnam Severance Hospital
ClinicalTrials.gov Identifier: NCT04520022    
Other Study ID Numbers: 3-2015-0285
First Posted: August 20, 2020    Key Record Dates
Last Update Posted: August 20, 2020
Last Verified: August 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sang Eun Lee, Gangnam Severance Hospital:
umbilical cord blood-derived mesenchymal stem cell
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
 Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Connective Tissue Diseases