Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01033552|
Recruitment Status : Recruiting
First Posted : December 16, 2009
Last Update Posted : November 13, 2019
|Condition or disease||Intervention/treatment||Phase|
|Epidermolysis Bullosa||Drug: Cyclophosphamide Drug: Fludarabine 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||Phase 2|
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, structural 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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||75 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||MT2009-09: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation and "Off-the-shelf" Mesenchymal Stem Cells|
|Actual Study Start Date :||January 2010|
|Estimated Primary Completion Date :||October 2021|
|Estimated Study Completion Date :||October 2021|
|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: Cytoxan
40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2.
Other Name: Fludara
Drug: Anti-thymocyte globulin
30 mg/kg on Days -4, -3 and -2.
Other Name: ATG
Drug: 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: CSA
Drug: 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: MSCT
Radiation: 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
- Event-free survival rate [ Time Frame: 1 year and 2 Years Post-transplant ]Event-free survival rate, with an event defined as death or failure to have a demonstrable increase in collagen, laminin, intergrin, keratin or plakin deposition.
- Transplant-related mortality (TRM) [ Time Frame: 180 Days Post Transplant ]Incidence of transplant-related mortality (TRM)
- Pattern of biochemical improvement [ Time Frame: Through 1 Year Post-Transplant ]Describe pattern of biochemical improvement as measured by an increase in protein expression (collagen, laminin, integrin, keratin, or plakin) and related structural and physical changes
- Quality of Life [ Time Frame: Pretreatment, Day 100, 6 months, 1 and 2 years ]Health quality of life questionnaire or iscorEB as compared to pretreatment results
- Durability of HSC and third party MSC engraftment in the skin [ Time Frame: 100 Days ]Incidence of HSC and third party MSC engraftment in the skin
- Probability of Survival [ Time Frame: 1 Year ]Number of surviving patients one year after engraftment
- Acute GVHD [ Time Frame: 100 Days ]Incidence of acute GCHD
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): NCT01033552
|Contact: Kim Nelson, RNfirstname.lastname@example.org|
|United States, Minnesota|
|University of Minnesota Masonic Cancer Center and Medical Center||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Kim Nelson, RN 612-273-2925 email@example.com|
|Principal Investigator: Jakub Tolar, MD, PhD|
|Principal Investigator:||Jakub Tolar, MD, PhD||Masonic Cancer Center, University of Minnesota|