Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

Terminated 7 enrolled Masonic Cancer Center, University of Minnesota

NaSingle-groupOpen-labelTreatment

Summary

RATIONALE: In animal models, stem cells have been shown to home to the skin and repair the biochemical and structural abnormalities associated with recessive dystrophic epidermolysis bullosa (RDEB) (collagen 7 deficiency). PURPOSE: To determine the safety and effectiveness of stem cell infusion in the treatment of RDEB.

Timeline

Start: 2007-04
Primary completion: 2011-08
Completion: 2011-08

Publications

Wagner JE, Ishida-Yamamoto A, McGrath JA, Hordinsky M, Keene DR, Woodley DT, Chen M, Riddle MJ, Osborn MJ, Lund T, Dolan M, Blazar BR, Tolar J. Bone marrow transplantation for recessive dystrophic epidermolysis bullosa. N Engl J Med. 2010 Aug 12;363(7):629-39. doi: 10.1056/NEJMoa0910501.

Drugs

Evaluation	Drug	Modality	Dose	Route
Background	Busulfan	Small molecule	0.8 mg/kg	Intravenous
Background	Busulfan	Small molecule	1.1 mg/kg	Intravenous
Background	Cyclophosphamide	Other / unclassified	50 mg/kg	Intravenous
Background	Fludarabine	Small molecule	25 mg/m2	Intravenous

Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

Summary

Timeline

Publications

Drugs

Indications