Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa
- Estimate the incidence of detectable donor-derived collagen type VII at day 100 in
patients with epidermolysis bullosa by donor.
- 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
- 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
PROJECTED ACCRUAL: 30 patients
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of Patients With Detectable Collagen Type VII
Number of patients with epidermolysis bullosa who had collagen type VII. Type VII collagen defects cause recessive dystrophic epidermolysis bullosa (RDEB), a blistering skin disorder often accompanied by epidermal cancers.
Day 100 Post Transplant
John E. Wagner, MD
Masonic Cancer Center, University of Minnesota
United States: Institutional Review Board
|Masonic Cancer Center, University of Minnesota||Minneapolis, Minnesota 55455|