Status:

COMPLETED

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

Lead Sponsor:

Gangnam Severance Hospital

Collaborating Sponsors:

Daewoong Pharmaceutical Co. LTD.

Conditions:

Recessive Dystrophic Epidermolysis Bullosa

Eligibility:

All Genders

10-60 years

Phase:

PHASE1

PHASE2

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 increa...

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 s...

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

October 13 2016

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

January 10 2020

Estimated Enrollment :

5 Patients enrolled

Trial Details

Trial ID

NCT04520022

Start Date

October 13 2016

End Date

January 10 2020

Last Update

August 20 2020

Active Locations (1)

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Page 1 of 1 (1 locations)

1

GangnamSeverance Hospital

Seoul, South Korea