Stem Cell Transplant for Inborn Errors of Metabolism

Phase 2/3 Completed 135 enrolled Masonic Cancer Center, University of Minnesota

Non-randomizedSingle-groupOpen-labelTreatment

Summary

The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic storage disease.

Timeline

Start: 1995-01
Primary completion: 2010-06
Completion: 2010-06

Publications

Miller WP, Rothman SM, Nascene D, Kivisto T, DeFor TE, Ziegler RS, Eisengart J, Leiser K, Raymond G, Lund TC, Tolar J, Orchard PJ. Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report. Blood. 2011 Aug 18;118(7):1971-8. doi: 10.1182/blood-2011-01-329235. Epub 2011 May 17.

Drugs

Evaluation	Drug	Modality	Dose	Route
Background	ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	Protein / enzyme biologic	15 mg/kg	Intravenous
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

Stem Cell Transplant for Inborn Errors of Metabolism

Summary

Timeline

Publications

Drugs

Indications