Quick Search (by title or disease)
  

Go to Advanced Search for more options.

Clinical Trials Help

Contact our Cancer Information Line nurse with your questions about open or upcoming clinical trials, second opinions, or patient referrals.
ccinfo@umn.edu • 612-624-2620 • 1-888-226-2376

  Sip Protocol

Masonic Cancer Research Center
ccinfo@umn.edu • 612-624-2620 • 1-888-226-2376

Back to Search Instructions

Protocol No. 2015LS076 Principal Investigator Tolar, Jakub
Phase Phase II
Age Group Both Scope Local
Secondary Protocol No. MT2015-20
Title MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions
Objective To estimate the event-free survival rate by 1 year post-transplant with an event defined as death or a 50% increase in a patients IScoreEB from baseline
Treatment Therapies involved - Hematopoietic Stem Cell Transplant and serial MSC Infusions Arm A - CYCLOPHOSPHAMIDE/FLUDARABINE/THYMOGLOBULIN/TOTAL BODY IRRADIATION Arm B – CYCLOPHOSPHAMIDE/FLUDARABINE/THYMOGLOBULIN/TOTAL BODY IRRADIATION /MESENCHYMAL STEM CELLS Arm C - CYCLOPHOSPHAMIDE/FLUDARABINE/THYMOGLOBULIN/TOTAL BODY IRRADIATION /MESENCHYMAL STEM CELLS
Description This is a single-institution, phase II study to determine the event-free survival at 1 year post allogeneic transplant and serial mesenchymal stem cell (MSC) infusions from a related donor (HLA identical, mismatched or haploidentical) or matched unrelated donor for the biochemical correction of severe epidermolysis bullosa (EB). A single marrow harvest is performed to collect the stem cells for the transplant procedure and, with the donor’s consent, an additional 25-35 ml sample for MSC production for post-transplant infusion on day 60, 100, and 180. The patient will be enrolled to either: Arm A: stem cell transplant alone Arm B: stem cell transplant plus serial MSC infusions Arm C: stem cell transplant plus serial MSC infusions (in the event of graft failure for a retransplant)
Key Eligibility Diagnosis of severe form of EB with documented collagen, laminin, integrin, keratin or plakin deficiency. 0- 25 years of age. An available healthy HLA matched or partially HLA matched related or unrelated donor for HSC graft and MSC Adequate renal, hepatic, cardiac and pulmonary function
Applicable Disease Sites Non Cancer
Drugs Involved Anti-Thymocyte Globulin - Rabbit (ATG)
CYCLOPHOSPHAMIDE (CYTOXAN, NEOSAR)
FLUDARABINE (FLUDARA)
Mesenchymal Stem Cells (MSC)
THYMOGLOBULIN
Total Body irradiation (TBI)
Status Open
Participating Institutions Masonic Cancer Center
University of Minnesota
Detailed Eligibility Inclusion Criteria • 0 through 25 years of age • Diagnosis of severe form of EB with documented collagen, laminin, integrin, keratin or plakin deficiency (by immunofluorescence staining with protein specific antibodies or Western blotting or by mutation analysis) • Adequate organ function within 4 weeks of study registration defined as: - Renal: glomerular filtration rate within normal range for age - Hepatic: bilirubin, AST/ALT, ALP < 5 x upper limit of normal - Pulmonary: oxygen saturation >92% - Cardiac: left ventricular ejection fraction &#8805; 45%, normal EKG or approved by Cardiology for transplant • Sexually active participants must agree to use adequate birth control for the during the study period (from before the start of the preparative chemotherapy through 1 year post-transplant) • Available donor per to section 5 • Voluntary written consent – adult or parent (with information sheet for minors, if applicable) prior to any research related procedures or treatment Exclusion Criteria • Active untreated systemic infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days) • History of HIV infection • Prior transplantation with donor skin • Evidence of squamous cell carcinoma • Pregnant or breast feeding. Females of child-bearing potential must have a negative pregnancy test prior to study registration as the agents administered in this study are Pregnancy Category C and D.
Treatment Type Non-Cancer Therapeutic
Contact Jakub Tolar Phone:612-624-7180
Email:tolar003@umn.edu