Stem Cell Transplantation for Hurler
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|ClinicalTrials.gov Identifier: NCT00176917|
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : September 4, 2009
Last Update Posted : December 28, 2017
|Condition or disease||Intervention/treatment||Phase|
|Mucopolysaccharidosis I Mucopolysaccharidosis VI Mannosidosis Mucolipidosis Type II (I-cell Disease)||Procedure: Stem Cell Transplant Drug: Busulfan, Cyclophosphamide, ATG||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||41 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis)|
|Study Start Date :||May 1999|
|Actual Primary Completion Date :||May 2008|
|Actual Study Completion Date :||May 2010|
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 Transplant
Drug: 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
- 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 ]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 ]Number of patients surviving (alive) at specified timepoints.
- Number of Patients Who Failed Engraftment. [ Time Frame: Day 42 Post Transplant ]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 ]Toxicity (undesireable effect) of this stem cell transplant preparative regimen due to acute graft-versus-host disease.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00176917
|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|