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

Phase 2 Completed 17 enrolled Masonic Cancer Center, University of Minnesota

Non-randomizedParallel-groupOpen-labelTreatment

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).

Timeline

Start: 2016-03
Primary completion: 2022-11-15
Completion: 2023-07-26

Publications

Ebens CL, McGrath JA, Tamai K, Hovnanian A, Wagner JE, Riddle MJ, Keene DR, DeFor TE, Tryon R, Chen M, Woodley DT, Hook K, Tolar J. Bone marrow transplant with post-transplant cyclophosphamide for recessive dystrophic epidermolysis bullosa expands the related donor pool and permits tolerance of nonhaematopoietic cellular grafts. Br J Dermatol. 2019 Dec;181(6):1238-1246. doi: 10.1111/bjd.17858. Epub 2019 Jun 28.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Busulfan	Small molecule	—	Intravenous
Subject	Donor mesenchymal stem cell infusions	Cell therapy	—	—
Background	Cyclophosphamide	Other / unclassified	50 mg/kg	Intravenous
Background	Fludarabine	Small molecule	30 mg/m2	Intravenous
Background	Mycophenolate Mofetil	Small molecule	15 mg/kg	Intravenous
Background	Tacrolimus	Small molecule	—	—
Background	Thymoglobulin	Protein / enzyme biologic	2 mg/kg	Intravenous

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

Summary

Timeline

Publications

Drugs

Indications