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Stem Cell Transplantation for Hurler

NCT00176917 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 41

Last updated 2017-12-28

Study results available
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Summary

The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for Hurler syndrome, Maroteaux Lamy syndrome, Mannosidosis, or I-cell disease.

Conditions

  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis VI
  • Mannosidosis
  • Mucolipidosis 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.

Sponsors & Collaborators

  • Masonic Cancer Center, University of Minnesota

    lead OTHER

Principal Investigators

  • Paul Orchard, MD · Masonic Cancer Center, University of Minnesota

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
1999-05-31
Primary Completion
2008-05-31
Completion
2010-05-31

Countries

  • United States

Study Locations

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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 NCT00176917 on ClinicalTrials.gov