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

NCT04520022 | Completed Phase 1

• 1 other identifier: 3-2015-0285
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Keywords

umbilical cord blood-derived mesenchymal stem cell

Outcome Measures

Primary Outcomes (1)

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

  8 months

Secondary Outcomes (8)

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

  baseline, day 56

• Change in Birmingham Epidermolysis Bullosa Severity Score (BEBSS)

  baseline, day56, day 112, day168

• Change in Global severity score

  baseline, day56, day 112, day168

• Change in total body surface area affected by RDEB

  baseline, day56, day 112, day168

• Change in Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire

  baseline, day56, day 112, day168

Study Arms (1)

FURESTEM-CD Inj

EXPERIMENTAL

Drug: Human Umbilical Cord Blood-derived Mesenchymal Stem Cells

Interventions

Human Umbilical Cord Blood-derived Mesenchymal Stem Cells DRUG

3.0 x 106 cells/kg, IV, Total of 3 doses every 2weeks

Also known as: hUCB-MSCs, FURESTEM-CD Inj

FURESTEM-CD Inj

Eligibility Criteria

• Age: 10 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Gangnam Severance Hospital: lead
• Daewoong Pharmaceutical Co. LTD.: collaborator

Study Sites (1)

GangnamSeverance Hospital

Seoul, South Korea

Location

Related Publications (1)

• 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.

  PMID: 33491668 DERIVED

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant professor

Study Record Dates

• First Submitted: August 13, 2020
• First Posted: August 20, 2020
• Study Start: October 13, 2016
• Primary Completion: January 10, 2020
• Study Completion: January 10, 2020
• Last Updated: August 20, 2020

Record last verified: 2020-08

Locations

KR (1)