Stem Cell Transplant for Epidermolysis Bullosa
Drug: Anti-thymocyte globulin
Drug: Cyclosporine A
Drug: Mycophenolate mofetil
Procedure: Mesenchymal stem cell transplantation
Radiation: Total body irradiation
Procedure: Bone marrow or umbilical cord blood (UCG) stem cell transplantation
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation|
- Event-free survival [ Time Frame: 1 year and 2 Years Post-transplant ] [ Designated as safety issue: No ]
- Incidence of transplant-related mortality (TRM) [ Time Frame: 180 Days Post Transplant ] [ Designated as safety issue: Yes ]
- Pattern of biochemical improvement measured by increase in protein expression (collagen, laminin, integrin or plakin) [ Time Frame: Through 1 Year Post-Transplant ] [ Designated as safety issue: No ]
- Quality of Life [ Time Frame: Pretreatment, Day 365 and Day 730 ] [ Designated as safety issue: No ]
|Study Start Date:||January 2010|
|Estimated Study Completion Date:||October 2019|
|Estimated Primary Completion Date:||October 2019 (Final data collection date for primary outcome measure)|
|Experimental: Transplant in Epidermolysis Bullosa||
Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Other Name: CytoxanDrug: Fludarabine
40 mg/m^2/day intravenously on Days -5, -4, -3 and -2.
Other Name: FludaraDrug: Anti-thymocyte globulin
30 mg/kg on Days -4, -3 and -2.
Other Name: ATGDrug: Cyclosporine A
Days -3 to 100+ to maintain a level of >200 ng/ml; initial dose 2.5 mg/kg over 2 hours every 8-12 hours for children.
Other Name: CSADrug: Mycophenolate mofetil
15 mg/kg intravenous twice per day on days -3 through 30.
Other Name: CellCept(R)Procedure: Mesenchymal stem cell transplantation
infused via intravenous drip on Day 0
Other Name: MSCTRadiation: Total body irradiation
300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.Procedure: Bone marrow or umbilical cord blood (UCG) stem cell transplantation
Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
Other Name: UCBSCT
The primary objective of this study is to estimate the event-free survival rate by 1 year post-transplant with an event defined as a death or failure to have a demonstrable increase in collagen, laminin, integrin, keratin or plakin deposition by 1 year post-transplant or other biochemical, structual or physical measure of improvement.
The secondary objectives of this study are to i) determine the incidence of transplant-related mortality (TRM) at 180 days; ii) describe the pattern of biochemical improvement as measured by an increase in protein expression (collagen, laminin, integrin, keratin or plakin) and related structural and physical changes; iii) describe health quality of life at day 365 and 730 as compared to pretreatment results; iv) describe the pattern and durability of HSC and third party MSC engraftment in the skin; v) determine the probability of survival at 1 year.
Patients with severe epidermolysis bullosa will be screened to meet the eligibility requirements, related or unrelated donor marrow or UCB will be infused, and subjects will be followed for a minimum of 5 years after stem cell transplant. A target accrual of 75 subjects over 5 years will be recruited to the study.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01033552
|Contact: Patricia Kleinke, RNfirstname.lastname@example.org|
|United States, Minnesota|
|University of Minnesota Masonic Cancer Center and Medical Center||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Patricia Kleinke, RN 612-273-0857 email@example.com|
|Principal Investigator: John E Wagner, MD|
|Principal Investigator:||Jakub Tolar, MD, PhD||Masonic Cancer Center, University of Minnesota|