Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Phase 2 Terminated 18 enrolled Masonic Cancer Center, University of Minnesota

NaSingle-groupOpen-labelTreatment

Summary

The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell transplantation (HCT).

Timeline

Start: 2008-03
Primary completion: 2010-02
Completion: 2010-02

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	Alemtuzumab	Monoclonal antibody	—	—
Background	Busulfan	Small molecule	—	—
Background	Cyclophosphamide	Other / unclassified	—	—

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Summary

Timeline

Publications

Drugs

Indications