Stem Cell Transplantation for Hurler

Completed · Phase 2

Conditions studied: Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)

In brief

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.

Key facts

Study ID

NCT00176917

Run by

Masonic Cancer Center, University of Minnesota

People needed

41

Starts

1999-05-01

Expected to finish

2010-05-01

Last updated by the study team

2017-12-28

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Masonic Cancer Center, University of Minnesota — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

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