Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa
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|ClinicalTrials.gov Identifier: NCT00478244|
Recruitment Status : Terminated (Competing studies)
First Posted : May 24, 2007
Results First Posted : February 11, 2013
Last Update Posted : December 28, 2017
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.
|Condition or disease||Intervention/treatment||Phase|
|Epidermolysis Bullosa||Drug: busulfan Drug: cyclophosphamide Drug: fludarabine phosphate Procedure: hematopoietic bone marrow transplantation||Not Applicable|
- 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 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 (Clinical Trial)|
|Actual Enrollment :||7 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa|
|Study Start Date :||April 2007|
|Actual Primary Completion Date :||August 2011|
|Actual Study Completion Date :||August 2011|
Experimental: Epidermolysis Bullosa (EB) Patients
Epidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant.
Day -9 through Day -6: 1.1 mg/kg if < 12 kg IV every 6 hours; 0.8 mg/kg if > 12 kg.
Other Name: BulsulfexDrug: cyclophosphamide
Day -5 through Day -2: 50 mg/kg IV over 120 min.
Other Name: CytoxanDrug: fludarabine phosphate
Day -5 through Day -3: 25 mg/m2 IV over 60 min.
Other Names:Procedure: hematopoietic bone marrow transplantation
allogeneic bone marrow, peripheral stem cell or umbilical cord blood transplantation
Other Name: Bone marrow transplant
- Number of Patients With Detectable Collagen Type VII [ Time Frame: Day 100 Post Transplant ]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.
- Number of Patients With >70% Donor Chimerism [ Time Frame: Days 21, 100, 180, 365 and 730 Post Transplant ]Number of patients with donor chimerism - percentage of donor cells in the patient via the peripheral blood or bone marrow.
- Number of Patients With Transplant-Related Mortality [ Time Frame: Day 180 Post Transplant ]Number of patients who died due to complications of the transplant (includes all deaths without previous relapse or progression).
- Number of Patients With Platelet Engraftment [ Time Frame: Day 180 Post Transplant ]Number of patients with a platelet count >5 x 10^10 cells/liter for 3 consecutive measurements.
- Number of Patients With Acute Graft-Versus-Host Disease (GVHD) [ Time Frame: Day 100 Post Transplant ]Number of patients with GVHD. Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
- Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD) [ Time Frame: Day 365 Post Transplant ]Number of patients with cGVHD; a severe long-term complication created by infusion of donor cells into a foreign host.
- Overall Survival [ Time Frame: 1 year and 2 years Post Transplant ]Survival is defined as the number of patients that were alive post transplant.
- Number of Patients With Donor Derived Cells in Skin [ Time Frame: Day 90 Post Transplant ]Number of patients who had donor skin chimerism - donor cells in the patient's epidermis (a state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease).
- Number of Patients With Resistance to Blister Formation [ Time Frame: Month 1 through Month 24 Inclusive ]Resistance to Blister Formation demonstrated by response to negative pressure.
- Number of Patients With Neutrophil Engraftment [ Time Frame: Day 42 Post Transplant ]Number of patients with an absolute neutrophil count >5 x 10^8 cells/liter for 3 consecutive days.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00478244
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota|
|Minneapolis, Minnesota, United States, 55455|
|Study Chair:||John E. Wagner, MD||Masonic Cancer Center, University of Minnesota|