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Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00010335
Recruitment Status : Completed
First Posted : February 2, 2001
Last Update Posted : March 6, 2015
Information provided by (Responsible Party):
Fred Hutchinson Cancer Research Center

Brief Summary:

OBJECTIVES: I. Determine the safety and long term complications of total body irradiation in combination with cyclophosphamide, anti-thymocyte globulin, and autologous CD34-selected peripheral blood stem cell (PBSC) transplantation in children with refractory autoimmune disorders.

II. Determine the efficacy of this treatment regimen in these patients. III. Determine the reconstitution of immunity after autologous CD34-selected PBSC transplantation in these patients.

IV. Determine engraftment of autologous CD34-selected PBSC in these patients.

Condition or disease Intervention/treatment Phase
Systemic Sclerosis Systemic Lupus Erythematosus Dermatomyositis Juvenile Rheumatoid Arthritis Autoimmune Diseases Procedure: Stem Cell Transplantation Device: CD34 selection Phase 1

Detailed Description:

PROTOCOL OUTLINE: This is a multicenter study. Patients receive filgrastim (G-CSF) subcutaneously daily until peripheral blood stem cell (PBSC) collection is completed. CD34+ cells are separated from the rest of the PBSCs.

Patients undergo total body irradiation twice daily on days -5 and -4. Patients receive anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5 and cyclophosphamide IV on days -3 and -2. CD34-selected PBSCs are reinfused on day 0. Patients receive G-CSF IV daily beginning on day 0 and continuing until blood counts recover.

Patients are followed annually for 5 years and then every 5 years thereafter.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study of High-Dose Immunosuppression Followed by Infusion of CD34-Selected Autologous or Syngeneic Peripheral Blood Stem Cells for Treatment of Refractory Autoimmune Disorders
Study Start Date : November 2000
Actual Primary Completion Date : July 2010
Actual Study Completion Date : May 2011

Arm Intervention/treatment
Experimental: 1
Participants will receive a stem cell transplant.
Procedure: Stem Cell Transplantation
Participants will receive a stem cell transplantation along with irradiation and the drugs anti-thymocyte globulin, cyclophosphamide, and filgrastim as noted in the text of this record.

Device: CD34 selection
CD34+ cells are separated from the rest of the peripheral blood stem cells.

Primary Outcome Measures :
  1. Mortality [ Time Frame: Annually for 5 years and then every 5 years thereafter ]

Secondary Outcome Measures :
  1. Immune reconstitution, engraftment, efficacy, late-effects [ Time Frame: Annually for 5 years and then every 5 years thereafter ]

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics--

  • Diagnosis of 1 of the following based on American College of Rheumatology (ACR) Criteria: Severe juvenile rheumatoid arthritis (systemic onset or polyarticular course) Juvenile systemic lupus erythematosus Systemic sclerosis Dermatomyositis
  • Refractory to standard or aggressive therapy OR unacceptable toxicity from standard therapy
  • Reasonable expectation of possible improvement as evidenced by a good potential for rehabilitation therapy and adequate social factors
  • No serious CNS damage that would preclude significant functional recovery

--Prior/Concurrent Therapy--

  • Chemotherapy: At least 4 weeks since prior methotrexate or cyclophosphamide
  • Endocrine therapy: At least 4 weeks since prior intra-arterial steroids Juvenile rheumatoid arthritis patients should continue steroids without taper throughout mobilization and harvest of stem cells If receiving corticosteroids, must be continued without taper


  • At least 4 weeks since prior anti-inflammatory agents such as non-steroidal anti-inflammatory drugs (NSAIDs) or sulfasalazine
  • At least 4 weeks since prior cyclosporine, tacrolimus, mycophenolate mofetil, azathioprine, penicillamine, or etanercept

--Patient Characteristics--

  • Life expectancy: At least 30 days
  • Hematopoietic: Absolute neutrophil count at least 1,000/mm3 OR Platelet count at least 100,000/mm3 No bone marrow aspirate or biopsy consistent with production defect (depletion of neutrophil precursors or megakaryocytes) No myelodysplasia
  • Hepatic: Bilirubin no greater than 2.5 mg/dL AST no greater than 300 U/L on two sequential tests No severe liver dysfunction within past month No active hepatitis A, B, or C
  • Renal: No end-stage glomerulonephritis or renal disease Creatinine clearance at least 40 mL/min
  • Cardiovascular: No uncontrolled malignant arrhythmia No New York Heart Association class III or IV congestive heart failure Ejection fraction at least 50%
  • Pulmonary: DLCO at least 45% (DLCO at least 70% for patients with pulmonary disease caused by documented processes other than primary autoimmune disorder, such as infectious pneumonia or aspiration pneumonia) No severe pulmonary hypertension (PAP greater than 50) without potential for significant improvement


  • No medical or psychosocial reasons that would make hematopoietic stem cell collection intolerable
  • No increased anesthetic risks
  • No fever higher than 39 degrees C
  • No positive serology for toxoplasmosis
  • No active life threatening infection not responsive to therapy
  • No other disease or organ dysfunction that would limit survival
  • No known hypersensitivity to murine or equine proteins
  • No known primary immunodeficiency disease HIV negative

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00010335

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United States, Washington
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109
Sponsors and Collaborators
Fred Hutchinson Cancer Research Center
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Study Chair: Ann Woolfrey Fred Hutchinson Cancer Research Center
Principal Investigator: Carol A. Wallace, MD Fred Hutchinson Cancer Research Center

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Responsible Party: Fred Hutchinson Cancer Research Center Identifier: NCT00010335     History of Changes
Other Study ID Numbers: 1353.00
199/15575 ( Other Identifier: ORD )
First Posted: February 2, 2001    Key Record Dates
Last Update Posted: March 6, 2015
Last Verified: March 2015
Keywords provided by Fred Hutchinson Cancer Research Center:
arthritis & connective tissue diseases
immunologic disorders and infectious disorders
juvenile rheumatoid arthritis
rare disease
systemic lupus erythematosus
systemic sclerosis
Additional relevant MeSH terms:
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Arthritis, Rheumatoid
Arthritis, Juvenile
Pathologic Processes
Lupus Erythematosus, Systemic
Scleroderma, Systemic
Scleroderma, Diffuse
Autoimmune Diseases
Joint Diseases
Musculoskeletal Diseases
Rheumatic Diseases
Connective Tissue Diseases
Immune System Diseases
Skin Diseases
Muscular Diseases
Neuromuscular Diseases
Nervous System Diseases
Antilymphocyte Serum
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action