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Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

NCT00668564 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2017-12-28

Study results available
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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).

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

Interventions

PROCEDURE

Stem Cell Transplantation

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.

DRUG

Cyclophosphamide

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

DRUG

Campath-1H

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

DRUG

Busulfan

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

Sponsors & Collaborators

  • Masonic Cancer Center, University of Minnesota

    lead OTHER

Principal Investigators

  • Paul Orchard, MD · University of Minnesota Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-03-31
Primary Completion
2010-02-28
Completion
2010-02-28

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00668564 on ClinicalTrials.gov