Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

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Brief Title

Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

Official Title

Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa

Brief Summary

      RATIONALE: In animal models, stem cells have been shown to home to the skin and repair the
      biochemical and structural abnormalities associated with recessive dystrophic epidermolysis
      bullosa (RDEB) (collagen 7 deficiency).

      PURPOSE: To determine the safety and effectiveness of stem cell infusion in the treatment of
      RDEB.
    

Detailed Description

      OBJECTIVES:

      Primary

        -  Estimate the incidence of detectable donor-derived collagen type VII at day 100 in
           patients with epidermolysis bullosa by donor.

      Secondary

        -  Determine the incidence of transplant-related mortality at day 180

        -  Determine the incidence of blood chimerism at days 21, 100, 180, 365, and 730

        -  Determine the incidence of neutrophil recovery at day 42 and platelet recovery at day
           180

        -  Determine the incidence of acute graft-versus-host disease (GVHD) grade II-IV and grade
           III-IV at day 100

        -  Determine the incidence of chronic GVHD at 1 year

        -  Determine the probability of survival at 1 and 2 years

        -  Determine the incidence of donor derived cells in the skin

        -  Determine resistance to blister formation OUTLINE: This is an open-label, pilot study.

        -  Conditioning regimen: Busulfan intravenously (IV) over 2 hours every 6 hours on days -9
           to -4, fludarabine phosphate IV over 1 hour on days -5 to -3, and high-dose
           cyclophosphamide IV over 1 hour on days -5 to -2.

        -  Stem cell transplantation on day 0.

      After completion of study treatment, patients are followed periodically for at least 5 years.

      PROJECTED ACCRUAL: 30 patients
    


Study Type

Interventional


Primary Outcome

Number of Patients With Detectable Collagen Type VII

Secondary Outcome

 Number of Patients With >70% Donor Chimerism

Condition

Epidermolysis Bullosa

Intervention

busulfan

Study Arms / Comparison Groups

 Epidermolysis Bullosa (EB) Patients
Description:  Epidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant.

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

7

Start Date

April 2007

Completion Date

August 2011

Primary Completion Date

August 2011

Eligibility Criteria

        Inclusion criteria:

          -  Diagnosis of epidermolysis bullosa (EB)

               -  Documented collagen type VII deficiency by:

                    -  Antigenic mapping (LH7.2 antibody)

                    -  Ultrastructure analysis of anchoring fibrils

                    -  DNA mutation analysis

          -  Performance status: >50% Lansky; >50% Karnofsky

          -  Adequate organ function

               -  Renal: glomerular filtration rate > 60ml/min/1.73m2 patients aged ≤ 10 years

               -  Hepatic: bilirubin, aspartate aminotransferase/alanine aminotransferase
                  (AST/ALT), Alkaline phosphatase (ALP) < 5 x upper limit of normal 4.2.3
                  Pulmonary: oxygen saturation >92% 4.2.4 Cardiac: left ventricular ejection
                  fraction > 45%.

          -  Healthy related hematopoietic stem cell donor available and meeting 1 of the following
             criteria:

               -  HLA-A, B, DRB1-identical sibling bone marrow and/or umbilical cord blood donor
                  (first priority)

               -  HLA-A, B, DRB1-matched or partially matched related donor (second priority)

               -  Donor may be a carrier but must be unaffected by EB

               -  8/8 HLA A, B, C, DRB1 allele level matched unrelated marrow donor (third
                  priority)

               -  7/8 HLA-A, B, C, DRB1 allele level matched unrelated marrow donor or 4/6 HLA-A, B
                  (antigen level), DRB1 (allele level) matched unrelated cord blood donor (fourth
                  priority)

        Exclusion criteria:

          -  Active infection at time of transplantation (including active infection with
             Aspergillus or other mold within 30 days)

          -  Squamous cell carcinoma of the skin

          -  History of human immunodeficiency virus (HIV) infection

          -  Prior transplantation with donor skin
      

Gender

All

Ages

N/A - 25 Years

Accepts Healthy Volunteers

No

Contacts

John E. Wagner, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00478244

Organization ID

MT2006-15

Secondary IDs

CDR0000546620

Responsible Party

Sponsor

Study Sponsor

Masonic Cancer Center, University of Minnesota


Study Sponsor

John E. Wagner, MD, Study Chair, Masonic Cancer Center, University of Minnesota


Verification Date

December 2017