Stem Cell Transplantation for Hurler (NCT00176917) | Clinical Trial Compass
CompletedPhase 2
Stem Cell Transplantation for Hurler
United States41 participantsStarted 1999-05
Plain-language 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.
Who can participate
SexALL
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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 med…
What they're measuring
1
Mean Percentage of Donor Cells in Study Population (Chimerism).
Timeframe: at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year
Trial details
NCT IDNCT00176917
SponsorMasonic Cancer Center, University of Minnesota