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