At a glance
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MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions
In Brief
A Phase 2 clinical trial evaluating Thymoglobulin, Cyclophosphamide, and 7 other interventions for Epidermolysis Bullosa. Completed, enrolled 17 participants across 1 site.
Detailed Summary
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).
Study Details
Timeline
Interventions
0.5 mg/kg IV over 6 hours on day -9 and 2 mg/kg IV over 4 hours on day -8 and day -7 with premeds and solumedrol through day -2
14.5 mg/kg IV over 1 hour day -6 and -5 50 mg/kg IV over 2 hours with mesna 40 mg/kg IV day +2 and +3
30 mg/m2 IV over 60 minutes days -6 through day -2
See arm description for dosing.
Bone marrow infusion on Day 0
Day +5 through day +100 with goals of 5-10 ug/L (not used for HLA-identical related donors). When used in non-MSD recipients, tapered over 6-8 weeks starting at day +100.
15 mg/kg IV q8h (based upon actual body weight) with the maximum total daily dose not to exceed 3 grams. Day +5 through day 35
Day 60, 100 and 180 (collected during donor BM harvest for graft)
busulfan IV over 3 hours on days -3 and -2 for HLA-mismatched BM recipients only (Arms F and G)