Stem Cell Transplantation for Hurler

PHASE2CompletedINTERVENTIONAL
Enrollment

41

Participants

Timeline

Start Date

May 31, 1999

Primary Completion Date

May 31, 2008

Study Completion Date

May 31, 2010

Conditions
Mucopolysaccharidosis IMucopolysaccharidosis VIMannosidosisMucolipidosis Type II (I-cell Disease)
Interventions
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).

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."

Trial Locations (1)

55455

Masonic Cancer Center, University of Minnesota, Minneapolis

All Listed Sponsors
lead

Masonic Cancer Center, University of Minnesota

OTHER

NCT00176917 - Stem Cell Transplantation for Hurler | Biotech Hunter | Biotech Hunter