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


Phase 2
N/A
N/A
Not Enrolling
Both
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)

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Trial Information

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


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.


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.

Type of Study:

Interventional

Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Mean Percentage of Donor Cells in Study Population (Chimerism).

Outcome Description:

Donor-derived engraftment determined by restriction fragment length polymorphism (RFLP).

Outcome Time Frame:

at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year

Safety Issue:

No

Principal Investigator

Paul Orchard, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Masonic Cancer Center, University of Minnesota

Authority:

United States: Institutional Review Board

Study ID:

UMN-MT1999-07

NCT ID:

NCT00176917

Start Date:

May 1999

Completion Date:

May 2010

Related Keywords:

  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis VI
  • Mannosidosis
  • Mucolipidosis Type II (I-cell Disease)
  • stem cell transplant
  • storage disease
  • errors of metabolism
  • Mucolipidoses
  • Mucopolysaccharidosis I
  • Alpha-Mannosidosis
  • Mannosidase Deficiency Diseases
  • Mucopolysaccharidoses
  • Mucopolysaccharidosis VI

Name

Location

Masonic Cancer Center, University of MinnesotaMinneapolis, Minnesota  55455