Status:
COMPLETED
Stem Cell Transplantation for Hurler
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Mucopolysaccharidosis I
Mucopolysaccharidosis VI
Eligibility:
All Genders
Phase:
PHASE2
Brief 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...
Detailed Description
Prior to transplantation, subjects will receive Busulfan intravenously (IV) via the Hickman line four times daily for 4 days, Cyclophosphamide intravenously via the Hickman line once a day for 4 days,...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
May 1 1999
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2010
Estimated Enrollment :
41 Patients enrolled
Trial Details
Trial ID
NCT00176917
Start Date
May 1 1999
End Date
May 1 2010
Last Update
December 28 2017
Active Locations (1)
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1
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States, 55455