Overview

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.