NCT02582775

MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs

MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 25 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Epidermolysis Bullosa

Interventions

Type	Name	Description
DRUG	Thymoglobulin	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
DRUG	Cyclophosphamide	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
DRUG	Fludarabine	30 mg/m2 IV over 60 minutes days -6 through day -2
RADIATION	Total Body Irradiation	See arm description for dosing.
PROCEDURE	Bone marrow infusion	Bone marrow infusion on Day 0
DRUG	Tacrolimus	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.
DRUG	Mycophenolate Mofetil	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
BIOLOGICAL	Donor mesenchymal stem cell infusions	Day 60, 100 and 180 (collected during donor BM harvest for graft)
DRUG	Busulfan	busulfan IV over 3 hours on days -3 and -2 for HLA-mismatched BM recipients only (Arms F and G)

Timeline

• Start date: 2016-03-01
• Primary completion: 2022-11-15
• Completion: 2023-07-26
• First posted: 2015-10-21
• Last updated: 2024-09-19
• Results posted: 2024-09-19

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study