Stem Cell Transplantation for Hurler
Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis)
Sponsor: Masonic Cancer Center, University of Minnesota
A PHASE2 clinical study on Mannosidosis and Mucolipidosis Type II (I-cell Disease), this trial is completed. The trial is conducted by Masonic Cancer Center, University of Minnesota and has accumulated 12 data snapshots since 1999. Longitudinal tracking of this trial contributes to a broader understanding of treatment development timelines.
Study Description(click to expand)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.
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.
Status Flow
Change History
12 versions recorded-
Sep 2024 — Present [monthly]
Completed PHASE2
-
Jul 2024 — Sep 2024 [monthly]
Completed PHASE2
-
Jan 2023 — Jul 2024 [monthly]
Completed PHASE2
-
Dec 2022 — Jan 2023 [monthly]
Completed PHASE2
-
Dec 2021 — Dec 2022 [monthly]
Completed PHASE2
▶ Show 7 earlier versions
-
Jan 2021 — Dec 2021 [monthly]
Completed PHASE2
-
Nov 2020 — Jan 2021 [monthly]
Completed PHASE2
-
Jun 2018 — Nov 2020 [monthly]
Completed PHASE2
-
May 2018 — Jun 2018 [monthly]
Completed PHASE2
-
Aug 2017 — May 2018 [monthly]
Completed PHASE2
-
Feb 2017 — Aug 2017 [monthly]
Completed PHASE2
-
Jan 2017 — Feb 2017 [monthly]
Completed PHASE2
First recorded
May 1999
Trial started
Per CT.gov start date — pre-dates our first snapshot
Eligibility Summary
No eligibility information available.
Contact Information
- Masonic Cancer Center, University of Minnesota
For direct contact, visit the study record on ClinicalTrials.gov .