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Pilot Study of Allogeneic Bone Marrow Transplantation Plus Cyclosporine and Mycophenolate Mofetil to Induce Mixed Hematopoietic Chimerism in Patients With Primary T-Cell Immunodeficiency Disorders - Article


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Fahr's Syndrome

Fahr//'s Syndrome


Clinical Trial: Pilot Study of Allogeneic Bone Marrow Transplantation Plus Cyclosporine and Mycophenolate Mofetil to Induce Mixed Hematopoietic Chimerism in Patients With Primary T-Cell Immunodeficiency Disorders

This study is currently recruiting patients.

Sponsored by: Fred Hutchinson Cancer Research Center
Information provided by: Office of Rare Diseases (ORD)

Purpose

OBJECTIVES: I. Determine the safety of cyclosporine and mycophenolate mofetil as a non-ablative conditioning and post-transplantation immunosuppression regimen in patients with primary T-cell immunodeficiency disorders who undergo HLA-matched related or unrelated bone marrow transplantation to induce mixed hematopoietic chimerism (establishment of 1-95% donor CD3+ cells). II. Determine the kinetics of immune reconstitution of lymphoid cell subsets, T-cell function, and B-cell function after allogeneic bone marrow transplantation in this patient population.

Condition Treatment or Intervention Phase
Purine-Pyrimidine Metabolism, Inborn Errors
Wiskott-Aldrich Syndrome
Bare Lymphocyte Syndrome
Lymphopenia
Job's Syndrome
DiGeorge Syndrome
Omenn syndrome
X-linked hyper IgM syndrome
Severe Combined Immunodeficiency
Immunologic Deficiency Syndromes
 Drug: cyclosporine
 Drug: mycophenolate mofetil
Phase I

MedlinePlus related topics:  Birth Defects;   Bleeding Disorders;   Blood and Blood Disorders;   Genetic Disorders;   Immune System and Disorders;   Lymphatic Diseases;   Metabolic Disorders
Genetics Home Reference related topics:  22q11.2 deletion syndrome

Study Type: Interventional
Study Design: Treatment

Further Study Details: 

Expected Total Enrollment:  12

Study start: November 2000

PROTOCOL OUTLINE: Patients are stratified according to type of primary T-cell immunodeficiency disorder (severe combined immunodeficiency syndrome (SCID) vs non-SCID) and donor status (related vs unrelated). All patients receive cyclosporine orally or IV on days -1 through 50 and oral mycophenolate mofetil on days 0 through 27 in the absence of unacceptable toxicity. Unrelated donor recipients and non-SCID patients also undergo total body irradiation on day 0. All patients then undergo allogeneic bone marrow transplantation on day 0. Cyclosporine taper regimen begins on day 50 and continues through day 180 unless evidence of graft-versus-host disease.

Eligibility

Ages Eligible for Study:  up to  55 Years,  Genders Eligible for Study:  Both

Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

  • Histologically proven primary T-cell immunodeficiency disorder
  • Diminished immune responses based on primary defect in T-cell immunity (deficiency in T-cell number or function) including the following diagnostic categories: Severe combined immunodeficiency syndromes (SCID): SCID with presence of B lymphocytes X-linked SCID. Autosomal recessive SCID. SCID with absence of T and B lymphocytes. Purine metabolite deficiency. Adenosine deaminase (ADA) deficiency. Purine nucleoside phosphorylase (PNP) deficiency. Other T-cell immunodeficiencies: Bare lymphocyte syndrome (BLS). Omenn's syndrome. Hyper IgM syndrome. Wiskott-Aldrich syndrome. DiGeorge syndrome. Functional T cell defects. Defects in T cell receptor-CD3 complex. Interleukin-2 deficiency. Zap70 defect. Other T-cell functional defects defined by assays or clinical syndrome No viral-associated T-cell immunodeficiency disorder (e.g., HIV). Ineligible for conventional therapy HLA-matched bone marrow donor available HLA genotypically identical OR HLA phenotypically identical unrelated Matched serologically at HLA-A and B, and molecularly at HLA-C, DRB1, and DQB1 (mismatch at HLA-C allowed if matched molecularly at HLA-A and B).

--Prior/Concurrent Therapy--

  • No concurrent growth factors with mycophenolate mofetil

--Patient Characteristics--

  • Age: 55 and under
  • Life expectancy: At least 30 days
  • Other: No other disease or organ dysfunction that would limit survival to less than 30 days

Location and Contact Information


Washington
      Fred Hutchinson Cancer Research Center, Seattle,  Washington,  98109,  United States; Recruiting
Ann Woolfrey  206-667-4453 

Study chairs or principal investigators

Ann Woolfrey,  Study Chair,  Fred Hutchinson Cancer Research Center   

More Information

Study ID Numbers:  199/13003; FHCRC-1227.00
Record last reviewed:  January 2005
Last Updated:  January 21, 2005
Record first received:  January 6, 2001
ClinicalTrials.gov Identifier:  NCT00008450
Health Authority: Unspecified
ClinicalTrials.gov processed this record on 2005-04-08


Source: ClinicalTrials.gov
Cache Date: April 9, 2005


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October 13, 2008



Page Updated: October 3, 2005
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