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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

This study is currently recruiting participants.
Verified by Office of Rare Diseases (ORD), September 2008

Sponsored by: Fred Hutchinson Cancer Research Center
Information provided by: Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier: NCT00008450
  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 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

Genetics Home Reference related topics:   22q11.2 deletion syndrome    aceruloplasminemia    adenosine deaminase deficiency    hemophilia    Job syndrome    L1 syndrome    thrombotic thrombocytopenic purpura    X-linked hyper IgM syndrome    X-linked severe combined immunodeficiency   

MedlinePlus related topics:   Bone Marrow Transplantation   

Drug Information available for:   Cyclosporine    Cyclosporin    Mycophenolate Mofetil    Mycophenolate mofetil hydrochloride    Adenosine   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment, Non-Randomized, Open Label, Parallel Assignment
Official Title:   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

Further study details as provided by Office of Rare Diseases (ORD):

Primary Outcome Measures:
  • Assessment of engraftment of donor T lymphocytes (1-95% donor CD3+ cells) [ Time Frame: 5 Years Post Transplant ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Kinetics of immune reconstitution following a non-lethal conditioning regimen in patients with immunodeficiency diseases [ Time Frame: 5 Years Post Transplant ] [ Designated as safety issue: No ]

Estimated Enrollment:   12
Study Start Date:   November 2000
Estimated Study Completion Date:   November 2014
Estimated Primary Completion Date:   November 2009 (Final data collection date for primary outcome measure)

Arms Assigned Interventions
1: Experimental
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. 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.
Drug: Cyclosporine
2 mg/kg/dose (every 8 hours, less than 6kg) or 2 mg/kg/dose (every 12 hours, more than 6 kg) from day - 3 to day +100, followed by taper to day 180
Drug: Mycophenolate mofetil
15 mg/kg/dose every 8 hours until day 40, then taper until day +96
2: Experimental
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.
Drug: Cyclosporine
2 mg/kg/dose (every 8 hours, less than 6kg) or 2 mg/kg/dose (every 12 hours, more than 6 kg) from day - 3 to day +100, followed by taper to day 180
Drug: Mycophenolate mofetil
15 mg/kg/dose every 8 hours until day 40, then taper until day +96

Detailed Description:

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
Accepts Healthy Volunteers:   No

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
  Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00008450

Locations
United States, Washington
Fred Hutchinson Cancer Research Center     Recruiting
      Seattle, Washington, United States, 98109
      Contact: Lauri Burroughs, MD         lburroug@fhcrc.org    

Sponsors and Collaborators
Fred Hutchinson Cancer Research Center

Investigators
Study Chair:     Lauri Burroughs, MD     Fred Hutchinson Cancer Research Center    
  More Information


Responsible Party:   Fred Hutchinson Cancer Research Center ( Lauri Burroughs, MD/ Primary Investigator )
Study ID Numbers:   199/13003, FHCRC-1227.00
First Received:   January 6, 2001
Last Updated:   September 2, 2008
ClinicalTrials.gov Identifier:   NCT00008450
Health Authority:   United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):
DiGeorge syndrome  
Job's syndrome  
Omenn syndrome  
Wiskott-Aldrich syndrome  
X-linked hyper IgM syndrome  
adenosine deaminase deficiency  
bare lymphocyte syndrome  
genetic diseases and dysmorphic syndromes  
hematologic disorders
immunologic disorders and infectious disorders
lymphopenia
primary immunodeficiency disease
purine nucleoside phosphorylase deficiency
rare disease
severe combined immunodeficiency

Study placed in the following topic categories:
Cyclosporine
Miconazole
Mycophenolic Acid
Leukocyte Disorders
Cyclosporins
Metabolism, Inborn Errors
Job's Syndrome
Wiskott-Aldrich Syndrome
Thrombocytopenia
Hemorrhagic Disorders
Hyper IgM syndrome
Mycophenolate mofetil
Hyperkinesis
Infant, Newborn, Diseases
DiGeorge syndrome
Purpura
Metabolic Diseases
Hematologic Diseases
Severe Combined Immunodeficiency
Blood Coagulation Disorders
Blood Platelet Disorders
Lymphopenia
Endocrine System Diseases
Omenn syndrome
Leukopenia
Metabolic disorder
Adenosine
Parathyroid Diseases
Blood Protein Disorders
Clotrimazole

Additional relevant MeSH terms:
Phagocyte Bactericidal Dysfunction
Anti-Infective Agents
Disease
Immunologic Factors
Molecular Mechanisms of Pharmacological Action
Immune System Diseases
Antineoplastic Agents
Physiological Effects of Drugs
DNA Repair-Deficiency Disorders
Enzyme Inhibitors
Antibiotics, Antineoplastic
Immunosuppressive Agents
Pharmacologic Actions
Pathologic Processes
Blood Coagulation Disorders, Inherited
Syndrome
Antifungal Agents
Therapeutic Uses
Dysgammaglobulinemia
Antirheumatic Agents
Dermatologic Agents

ClinicalTrials.gov processed this record on November 20, 2008




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