Stem Cell Transplantation for Hurler

• Status: Completed
• Trial end date: 2010-05-01
• Target enrollment: 0
• Participant gender: All

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 Hurler syndrome, Maroteaux Lamy syndrome, Mannosidosis, or I-cell disease.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota
• Treatments: Busulfan; Cyclophosphamide; Thymoglobulin

Criteria

Inclusion Criteria:

• Patients with Mucopolysaccharidosis, type I (e.g., Hurler syndrome), Maroteaux-Lamy syndrome (MPS VI), Alpha Mannosidosis, or mucolipidosis type II (I-cell disease) who have an HLA-identical or mismatched (at 1 antigen) related marrow, PBSC, or cord blood donor.

• Patients with Mucopolysaccharidosis, type I, Maroteaux-Lamy syndrome (MPS VI), Alpha Mannosidosis, or mucolipidosis type II (I-cell disease) who have an HLA-identical or HLA-1 antigen mismatched unrelated marrow, PBSC, or HLA-0-2 antigen mismatched umbilical cord blood donor.

• Patients with MPS type I, Maroteaux Lamy Syndrome (MPS VI), or mucolipidosis type II (I-cell disease) will have a mental developmental index within two standard deviations of the normal mean, as best as can be determined using Bayley scales of infant development or other standardized testing, recognizing that these may be affected by speech and/or hearing impairment.

• Adequate organ function:

• Cardiac: ejection fraction >40%; no decompensated congestive heart failure or uncontrolled arrhythmia

• Renal: serum creatinine <2.0 mg/dl

• Hepatic: total bilirubin <3x Upper limits of normal transaminases < 5.0 x Upper limits of normal

• Signed consent.

Exclusion Criteria:

• Presence of major organ dysfunction (see above)

• Pregnancy

• Evidence of HIV infection or known HIV positive serology

• Patients or parents are psychologically incapable of undergoing BMT with associated strict isolation or documented history of medical non-compliance

• Patients >50 kg may be at risk for having cell doses below the goal of ≥ 10 x 106 CD 34 cells/kg and therefore will not be eligible to receive unrelated PBSCs.