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Stem Cell Transplantation for Hurler

This study has been completed.

Study NCT00176917. Last updated on September 12, 2008. Information provided by University of Minnesota

This Tabular View shows the required WHO registration data elements as marked by ^†

Descriptive Information Fields

Brief Title ^†

Stem Cell Transplantation for Hurler

Official Title ^†

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

Brief 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.

Study Phase

Phase II, Phase III

Study Type ^†

Interventional

Study Design ^†

Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study

Primary Outcome Measure ^†

estimate donor-derived engraftment by RFLP and a-L-iduronidase enzyme (Hurler syndrome), arylsulfatase B (Maroteaux Lamy syndrome), or Alpha Mannosidase (Mannosidosis) enzyme activity levels [ Time Frame: at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year ] [ Designated as safety issue: No ]

Secondary Outcome Measure ^†

estimate survival . [ Time Frame: at 100 days, 1 year, and 3 years post transplant ] [ Designated as safety issue: No ]

Condition ^†

Mucopolysaccharidosis I
Mucopolysaccharidosis VI
Mannosidosis
Mucolipidosis Type II (I-Cell Disease)

Intervention ^†

Procedure: Stem Cell Transplant
Drug: Busulfan, Cyclophosphamide, ATG

MEDLINE PMIDs

Links

Recruitment Information Fields

Recruitment Status ^†

Completed

Enrollment ^†

Start Date ^†

May 1999

Completion Date

March 2008

Eligibility Criteria ^†

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.

Gender

Both

Ages

Accepts Healthy Volunteers

Contacts ^††

Location Countries ^†

United States

Administrative Information Fields

NCT ID ^†

NCT00176917

Organization ID

0104M93821

Secondary IDs ^††

MT1999-07

Study Sponsor ^†

Orchard, Paul J., MD

Collaborators ^††

Masonic Cancer Center, University of Minnesota

Investigators ^†

Principal Investigator:

Paul Orchard, MD

Masonic Cancer Center, University of Minnesota

Information Provided By

University of Minnesota

Verification Date

September 2008

First Received Date ^†

September 12, 2005

Last Updated Date

September 12, 2008

^† Required WHO trial registration data element.
^†† WHO trial registration data element that is required only if it exists.