Stem Cell Transplantation for Hurler
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.
Mucolipidosis Type II (I-cell Disease)
Procedure: Stem Cell Transplant
Drug: Busulfan, Cyclophosphamide, ATG
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis)|
- Mean Percentage of Donor Cells in Study Population (Chimerism). [ Time Frame: at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year ] [ Designated as safety issue: No ]Donor-derived engraftment determined by restriction fragment length polymorphism (RFLP).
- Number of Patients Surviving on Study [ Time Frame: at 100 days, 1 year, and 3 years post transplant ] [ Designated as safety issue: Yes ]Number of patients surviving (alive) at specified timepoints.
- Number of Patients Who Failed Engraftment. [ Time Frame: Day 42 Post Transplant ] [ Designated as safety issue: Yes ]Toxicity (undesireable effect) of hematologic donor cell engraftment is determined by failure to engraft at Day 42.
- Number of Patients With Grade III-IV Acute Graft-versus-host Disease (aGVHD). [ Time Frame: Day 100 Post Transplant ] [ Designated as safety issue: Yes ]Toxicity (undesireable effect) of this stem cell transplant preparative regimen due to acute graft-versus-host disease.
|Study Start Date:||May 1999|
|Study Completion Date:||May 2010|
|Primary Completion Date:||May 2008 (Final data collection date for primary outcome measure)|
Experimental: Treatment Arm
All patients treated with chemotherapy and transplantation.
Procedure: Stem Cell Transplant
The purpose of hematopoietic cell transplantation is to introduce hematopoietic cells from a normal donor that contains the enzyme able to get rid of the substances that have accumulated in the body of patients with storage diseases. Hematopoietic cells can come from bone marrow, peripheral blood (i.e., the blood circulating in our body's blood vessels) or umbilical cord blood (i.e. blood taken from the umbilical cord after a baby is born and umbilical cord is cut).
Other Name: Bone Marrow TransplantDrug: Busulfan, Cyclophosphamide, ATG
Prior to transplantation, subjects will receive BUSULFAN intravenously (IV) via the Hickman line twice 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 help the new marrow "take" and grow. METHYLPREDNISOLONE will be given as a pre-medication for the ATG.
Other Name: Busulfex, Cytoxan, Thymoglobulin
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.
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota|
|Minneapolis, Minnesota, United States, 55455|
|Principal Investigator:||Paul Orchard, MD||Masonic Cancer Center, University of Minnesota|