Clinical trial NCT00668564
Conditions: Hurler's Syndrome Maroteaux-Lamy Syndrome Sly Syndrome Alpha Mannosidosis Fucosidosis Aspartylglucosaminuria Sphingolipidoses Krabbe Disease Wolman's Disease Niemann-Pick Disease Type B Niemann-Pick Disease, Type C
Keywords: Inborn errors of metabolism Sphingolipidoses Recessive Leukodystrophies- GLD, Krabbe disease, MLD Peroxisomal Disorders Wolman syndrome Niemann-Pick B patients Niemann-Pick C subtype 2
Sponsor: Masonic Cancer Center, University of Minnesota
Start date: 2008-03-01; Completion date: 2010-02-01
Enrollment: 18 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell transplantation (HCT).

Detailed description:
This has been an ongoing area of interest by our group at the Univ. of Minnesota, but this is a new protocol to take the place of several older protocols. While survival has been very good on the prior protocols over the past decade, incomplete engraftment has remained somewhat problematic. Therefore, we have modified the preparative regimen somewhat to increase engraftment by replacing anti-thymocyte globulin (ATG) with Campath-1H, a drug that is more immune suppressive. In addition, we have modified the supportive care regimen. Based on this, we will monitor levels of an anti-oxidant therapy (N-acetylcysteine) and biomarkers of inflammation and oxidative stress for the families that consent to these research studies.

Arms: arm 1: All patients treated with study regimen.
Interventions: intervention 1: Stem Cell Transplantation intervention 2: Cyclophosphamide intervention 3: Campath-1H intervention 4: Busulfan
Intervention descriptions: intervention 1: The purpose of hematopoietic stem cell transplantation is to introduce blood producing cells from a normal donor. These cells can either provide what is missing in the body to the other cells, or can change the body's immune response to the substances that have accumulated in the body. These normal hematopoietic stem 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). The new donor cells repopulate the blood and bone marrow system and enter the organs of the body, including the brain. Wherever these cells go, they will produce the needed enzyme. intervention 2: Days before Transplant Drug Frequency

* 4 Cyclophosphamide Once, given over 2 hours
* 3 Cyclophosphamide Once, given over 2 hours
* 2 Cyclophosphamide Once, given over 2 hours
* 1 Cyclophosphamide Once, given over 2 hours intervention 3: Days before Transplant Drug Frequency

12 Campath-1H Once, given over 2 hours

11 Campath-1H Once, given over 2 hours

10 Campath-1H Once, given over 2 hours intervention 4: Days before Transplant Drug Frequency

9 Busulfan Four times per day

8 Busulfan Four times per day

7 Busulfan Four times per day

6 Busulfan Four times per day

Protocol documents: