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Stem Cell Transplantation for Hurler | Clinical Trials | Clareo Health

COMPLETEDPHASE2

Stem Cell Transplantation for Hurler

Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis)

NCT00176917•Masonic Cancer Center, University of Minnesota

View on ClinicalTrials.gov

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.

Detailed Description

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.

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Patients with Mucopolysaccharidosis, type I (e.g., Hurler syndrome), Maroteaux-Lamy syndrome (MPS VI), Alpha Mannosidosis, or mucolipidosis type II (I-cell disease) who have an HLA-identical or mismatched (at 1 antigen) related marrow, PBSC, or cord blood donor.
•Patients with Mucopolysaccharidosis, type I, Maroteaux-Lamy syndrome (MPS VI), Alpha Mannosidosis, or mucolipidosis type II (I-cell disease) who have an HLA-identical or HLA-1 antigen mismatched unrelated marrow, PBSC, or HLA-0-2 antigen mismatched umbilical cord blood donor.
•Patients with MPS type I, Maroteaux Lamy Syndrome (MPS VI), or mucolipidosis type II (I-cell disease) will have a mental developmental index within two standard deviations of the normal mean, as best as can be determined using Bayley scales of infant development or other standardized testing, recognizing that these may be affected by speech and/or hearing impairment.
•Adequate organ function:
•Cardiac: ejection fraction \>40%; no decompensated congestive heart failure or uncontrolled arrhythmia
•Renal: serum creatinine \<2.0 mg/dl
•Hepatic: total bilirubin \<3x Upper limits of normal transaminases \< 5.0 x Upper limits of normal
•Signed consent.

Exclusion Criteria

•Presence of major organ dysfunction (see above)
•Pregnancy
•Evidence of HIV infection or known HIV positive serology
•Patients or parents are psychologically incapable of undergoing BMT with associated strict isolation or documented history of medical non-compliance
•Patients \>50 kg may be at risk for having cell doses below the goal of ≥ 10 x 106 CD 34 cells/kg and therefore will not be eligible to receive unrelated PBSCs.

Locations (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Key Dates

Start Date

May 1, 1999

Primary Completion Date

May 1, 2008

Completion Date

May 1, 2010

Last Updated

December 28, 2017

Enrollment

ACTUAL participants

Conditions

Mucopolysaccharidosis IMucopolysaccharidosis VIMannosidosisMucolipidosis Type II (I-cell Disease)

Interventions

Stem Cell Transplant

PROCEDURE

Busulfan, Cyclophosphamide, ATG

DRUG

Mucopolysaccharidosis VII Disease Monitoring Program

NCT03604835

Mucopolysaccharidosis VIIMPS VII+2 more

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RECRUITINGPHASE1

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

NCT02254863

AdrenoleukodystrophyBatten Disease+10 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 28, 2017Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Stem Cell Transplantation for Hurler

Inclusion Criteria

Exclusion Criteria

Mucopolysaccharidosis VII Disease Monitoring Program

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

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