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

August 17, 2020 updated by: Sang Eun Lee, Gangnam Severance Hospital

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

5

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

8 years to 58 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

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: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Adverse events related to the intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell
Time Frame: 8 months
8 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in type VII collagen and anchoring fibril expression at dermoepidermal junction
Time Frame: baseline, day 56
baseline, day 56
Change in Birmingham Epidermolysis Bullosa Severity Score (BEBSS)
Time Frame: baseline, day56, day 112, day168
baseline, day56, day 112, day168
Change in Global severity score
Time Frame: baseline, day56, day 112, day168
baseline, day56, day 112, day168
Change in total body surface area affected by RDEB
Time Frame: baseline, day56, day 112, day168
baseline, day56, day 112, day168
Change in Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire
Time Frame: baseline, day56, day 112, day168
baseline, day56, day 112, day168
Change in blister count
Time Frame: baseline, day56, day 112, day168
baseline, day56, day 112, day168
Change in pruritus visual analogue scale (VAS)
Time Frame: baseline, day56, day 112, day168
baseline, day56, day 112, day168
Change in pain visual analogue scale (VAS)
Time Frame: baseline, day56, day 112, day168
baseline, day56, day 112, day168

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Gangnam Severance Hospital

Collaborators

Daewoong Pharmaceutical Co. LTD.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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)

October 13, 2016

Primary Completion (Actual)

January 10, 2020

Study Completion (Actual)

January 10, 2020

Study Registration Dates

First Submitted

August 13, 2020

First Submitted That Met QC Criteria

August 17, 2020

First Posted (Actual)

August 20, 2020

Study Record Updates

Last Update Posted (Actual)

August 20, 2020

Last Update Submitted That Met QC Criteria

August 17, 2020

Last Verified

August 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 3-2015-0285

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.

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