NCT00478244

Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

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

• Status: Terminated
• Phase: N/A
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 25 Years
• Healthy volunteers: Not accepted

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

Conditions

• Epidermolysis Bullosa

Interventions

Type	Name	Description
DRUG	busulfan	Day -9 through Day -6: 1.1 mg/kg if \< 12 kg IV every 6 hours; 0.8 mg/kg if \> 12 kg.
DRUG	cyclophosphamide	Day -5 through Day -2: 50 mg/kg IV over 120 min.
DRUG	fludarabine phosphate	Day -5 through Day -3: 25 mg/m2 IV over 60 min.
PROCEDURE	hematopoietic bone marrow transplantation	allogeneic bone marrow, peripheral stem cell or umbilical cord blood transplantation

Timeline

• Start date: 2007-04-01
• Primary completion: 2011-08-01
• Completion: 2011-08-01
• First posted: 2007-05-24
• Last updated: 2017-12-28
• Results posted: 2013-02-11

Locations

1 site across 1 country: United States