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| Descriptive Information Fields | |||||
| Brief Title † | Stem Cell Transplantation for Hurler | ||||
| Official Title † | Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis) | ||||
| 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 for Hurler syndrome, Maroteaux Lamy syndrome, Mannosidosis, or I-cell disease. |
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| 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, and Anti-Thymocyte Globulin IV via the Hickman line twice daily for three days before the transplant. These three drugs are being given to subjects to help the new marrow "take" and grow. On the day of transplantation, the donor's hematopoietic cells will be transfused via central venous catheter. After hematopoietic cell transplant, subjects will then receive two drugs, cyclosporin and either methylprednisolone or Mycophenolate Mofetil (MMF). Cyclosporin and methylprednisolone or MMF are given to help prevent the complication of graft-versus-host disease and to decrease the chance that the new donor cells will be rejected. |
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| Study Phase | Phase II, Phase III | ||||
| Study Type † | Interventional | ||||
| Study Design † | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study | ||||
| Primary Outcome Measure † | estimate donor-derived engraftment by RFLP and a-L-iduronidase enzyme (Hurler syndrome), arylsulfatase B (Maroteaux Lamy syndrome), or Alpha Mannosidase (Mannosidosis) enzyme activity levels [ Time Frame: at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year ] [ Designated as safety issue: No ] | ||||
| Secondary Outcome Measure † | estimate survival . [ Time Frame: at 100 days, 1 year, and 3 years post transplant ] [ Designated as safety issue: No ] | ||||
| Condition † | Mucopolysaccharidosis I Mucopolysaccharidosis VI Mannosidosis Mucolipidosis Type II (I-Cell Disease) |
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| Intervention † | Procedure: Stem Cell Transplant Drug: Busulfan, Cyclophosphamide, ATG |
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| MEDLINE PMIDs | |||||
| Links | |||||
| Recruitment Information Fields | |||||
| Recruitment Status † | Completed | ||||
| Enrollment † | 38 | ||||
| Start Date † | May 1999 | ||||
| Completion Date | March 2008 | ||||
| Eligibility Criteria † | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Both | ||||
| Ages | |||||
| Accepts Healthy Volunteers | No | ||||
| Contacts †† | |||||
| Location Countries † | United States | ||||
| Administrative Information Fields | |||||
| NCT ID † | NCT00176917 | ||||
| Organization ID | 0104M93821 | ||||
| Secondary IDs †† | MT1999-07 | ||||
| Study Sponsor † | Orchard, Paul J., MD | ||||
| Collaborators †† | Masonic Cancer Center, University of Minnesota | ||||
| Investigators † |
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| Information Provided By | University of Minnesota | ||||
| Verification Date | September 2008 | ||||
| First Received Date † | September 12, 2005 | ||||
| Last Updated Date | September 12, 2008 | ||||