Back to resultsSafety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB
Primary Purpose
Recessive Dystrophic Epidermolysis Bullosa
Status
Completed
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Human Umbilical Cord Blood-derived Mesenchymal Stem Cells
Sponsored by
About this trial
This is an interventional treatment trial for Recessive Dystrophic Epidermolysis Bullosa focused on measuring umbilical cord blood-derived mesenchymal stem cell
Eligibility Criteria
Inclusion Criteria:
- 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).
- 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))
- 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)
- Patients who can be monitored during a clinical trial period
Exclusion Criteria:
- Patients who disagree with this study
- Patients who is not accompanied by a guardian if those with impaired consent ability
- Patient or the patient's representative is unable to hear and understand the explanation
- In case of received immunotherapy or chemotherapy including oral corticosteroid (topical treatment is possible) for more than 1 week within 8 weeks before registration.
- All kinds of live vaccines except influenza vaccine within four weeks prior to registration
- Clinically significant infections within four weeks of the screening date or during the screening period (pneumonia, pyelonephritis, Clostridium difficile etc)
- All kinds of confirmed congenital or acquired immunodeficiency syndrome
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.)
- Patients who with allogenic stem cell treatment experience within 1 year from the screening test date
- Patients who have a history of malignant tumors or is currently being treated (squamous cell carcinoma of the skin, cutaneous squamous cell carcinoma inclusion)
- Type VII collagen ELISA positive and IIF positive
- 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.)
- Other cases where the researcher judges that participation in this clinical trial is inappropriate
- If other clinical trial drugs have been administered within 4 weeks prior to registration or are currently participating in a clinical trial
Sites / Locations
- GangnamSeverance Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
FURESTEM-CD Inj
Arm Description
Outcomes
Primary Outcome Measures
Adverse events related to the intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell
Secondary Outcome Measures
Change in type VII collagen and anchoring fibril expression at dermoepidermal junction
Change in Birmingham Epidermolysis Bullosa Severity Score (BEBSS)
Change in Global severity score
Change in total body surface area affected by RDEB
Change in Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire
Change in blister count
Change in pruritus visual analogue scale (VAS)
Change in pain visual analogue scale (VAS)
Full Information
NCT ID
NCT04520022
First Posted
August 13, 2020
Last Updated
August 17, 2020
Sponsor
Gangnam Severance Hospital
Collaborators
Daewoong Pharmaceutical Co. LTD.
1. Study Identification
Unique Protocol Identification Number
NCT04520022
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
Study Type
Interventional
2. Study Status
Record Verification Date
August 2020
Overall Recruitment Status
Completed
Study Start Date
October 13, 2016 (Actual)
Primary Completion Date
January 10, 2020 (Actual)
Study Completion Date
January 10, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Gangnam Severance Hospital
Collaborators
Daewoong Pharmaceutical Co. LTD.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Recessive Dystrophic Epidermolysis Bullosa
Keywords
umbilical cord blood-derived mesenchymal stem cell
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Actual)
8. Arms, Groups, and Interventions
Arm Title
FURESTEM-CD Inj
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Human Umbilical Cord Blood-derived Mesenchymal Stem Cells
Other Intervention Name(s)
hUCB-MSCs, FURESTEM-CD Inj
Intervention Description
3.0 x 106 cells/kg, IV, Total of 3 doses every 2weeks
Primary Outcome Measure Information:
Title
Adverse events related to the intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell
Time Frame
8 months
Secondary Outcome Measure Information:
Title
Change in type VII collagen and anchoring fibril expression at dermoepidermal junction
Time Frame
baseline, day 56
Title
Change in Birmingham Epidermolysis Bullosa Severity Score (BEBSS)
Time Frame
baseline, day56, day 112, day168
Title
Change in Global severity score
Time Frame
baseline, day56, day 112, day168
Title
Change in total body surface area affected by RDEB
Time Frame
baseline, day56, day 112, day168
Title
Change in Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire
Time Frame
baseline, day56, day 112, day168
Title
Change in blister count
Time Frame
baseline, day56, day 112, day168
Title
Change in pruritus visual analogue scale (VAS)
Time Frame
baseline, day56, day 112, day168
Title
Change in pain visual analogue scale (VAS)
Time Frame
baseline, day56, day 112, day168
10. Eligibility
Sex
All
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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).
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))
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)
Patients who can be monitored during a clinical trial period
Exclusion Criteria:
Patients who disagree with this study
Patients who is not accompanied by a guardian if those with impaired consent ability
Patient or the patient's representative is unable to hear and understand the explanation
In case of received immunotherapy or chemotherapy including oral corticosteroid (topical treatment is possible) for more than 1 week within 8 weeks before registration.
All kinds of live vaccines except influenza vaccine within four weeks prior to registration
Clinically significant infections within four weeks of the screening date or during the screening period (pneumonia, pyelonephritis, Clostridium difficile etc)
All kinds of confirmed congenital or acquired immunodeficiency syndrome
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.)
Patients who with allogenic stem cell treatment experience within 1 year from the screening test date
Patients who have a history of malignant tumors or is currently being treated (squamous cell carcinoma of the skin, cutaneous squamous cell carcinoma inclusion)
Type VII collagen ELISA positive and IIF positive
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.)
Other cases where the researcher judges that participation in this clinical trial is inappropriate
If other clinical trial drugs have been administered within 4 weeks prior to registration or are currently participating in a clinical trial
Facility Information:
Facility Name
GangnamSeverance Hospital
City
Seoul
Country
Korea, Republic of
12. IPD Sharing Statement
Citations:
PubMed Identifier
33491668
Citation
Lee SE, Lee SJ, Kim SE, Kim K, Cho B, Roh K, Kim SC. Intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa patients. JCI Insight. 2021 Jan 25;6(2):e143606. doi: 10.1172/jci.insight.143606.
Results Reference
derived
Learn more about this trial
Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB
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