Epidermolysis Bullosa Clinical Trial
Official title:
Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa
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.
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
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