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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 19 February 2015
Main ID:  NCT00006055
Date of registration: 05/07/2000
Prospective Registration: Yes
Primary sponsor: Fairview University Medical Center
Public title: Autologous Peripheral Blood Stem Cell Transplantation in Patients With Life Threatening Autoimmune Diseases
Scientific title:
Date of first enrolment: March 2000
Target sample size: 10
Recruitment status: Active, not recruiting
URL:  http://clinicaltrials.gov/show/NCT00006055
Study type:  Interventional
Study design:  Primary Purpose: Treatment  
Phase:  N/A
Countries of recruitment
United States
Contacts
Name:     Arne Slungaard
Address: 
Telephone:
Email:
Affiliation:  Fairview University Medical Center
Key inclusion & exclusion criteria

Autoimmune thrombocytopenia purpura: platelet count less than 20,000/mm3 Adequate or
increased marrow megakaryocytes Presence of detectable platelet associated immunoglobulins
not due to alloreactive antibodies or posttransfusion purpura Prior response to
immunosuppressive therapy Platelet count chronically less than 20,000/mm3 with petechial
bleeding or less than 50,000/mm3 with other bleeding OR Any history of life threatening
hemorrhage Refractory to conventional therapy for at least 21 days Splenectomy At least 1
additional immunosuppressive therapy applied after splenectomy OR Controlled on
conventional therapy but at price of unacceptable toxicity: Serious steroid related
toxicity Absolute neutrophil count less than 500/mm3 25% of time, pure red blood cell
transfusion dependent or other toxicities (e.g., hemorrhagic cystitis) that are a
consequence of chronic or cytotoxic therapy Unable to wean from chronic daily or
intermittent cytotoxic therapy

Autoimmune hemolytic anemia or pure red cell aplasia, AIHA: Hemolytic anemia Hemoglobin
less than 10.0 g/dL without transfusion Hemolysis as evidenced by both: Sustained
reticulocytosis (greater than 125,000/mm3) without evidence of active bleeding or
increasing hemoglobin Laboratory evidence of hemolysis Positive direct antiglobulin test
or equivalent immune adherence test No evidence for paroxysmal nocturnal hemoglobinuria
Negative Ham's test and sucrose hemolysis. For PRCA: Anemia due to selective decrease in
marrow erythroid precursors Hemoglobin less than 10.0 g/dL without transfusion Severe
reticulocytopenia (less than 20,000/mm3 despite anemia) Severely decreased marrow
erythroid precursors Positive marrow coculture with serum or cells or response to
immunosuppression No evidence for PNH Negative Ham's test and sucrose hemolysis Severe
disease: Chronic (i.e., greater than 1 year) Transfusion dependent or untransfused
hemoglobin less than 8.0 g/dL Ferritin greater than 2,000 or evidence of organ dysfunction
due to iron overload Refractory to conventional therapy after all 3 of the following:
High dose steroids (at least 1 mg/kg) for at least 21 days Splenectomy (except cold
reactive antibodies) 1 additional immunosuppressive therapy OR Controlled on conventional
therapy but at price of unacceptable toxicity

Rheumatoid arthritis: Morning stiffness for at least 6 weeks Arthritis of 3 or more joint
areas Arthritis of hand joints Symmetric arthritis Rheumatoid nodules Serum rheumatoid
factor Radiographic changes Active rheumatoid disease as evidenced by all of the
following: Elevated Westergren erythrocyte sedimentation rate Minimum of 16 swollen or
tender joints using the 28 joint count method Must be at high risk for developing
deforming joint disease as defined by at least 2 of the following: High titer IgM-IgG
rheumatoid factor Radiographic evidence of erosive arthritis developing within the first
24 months of clinical disease Functional class II or III Refractory to conventional
therapy after 12 months of: Methotrexate used in combination with cyclosporine,
hydroxychloroquine, or sulfasalazine OR Intramuscular gold therapy (total dose greater
than 1.0 g and duration at least 6 months) OR Controlled on conventional therapy but at
price of unacceptable toxicity

Juvenile rheumatoid arthritis: Under 16 years of age at onset Arthritis in 1 or more
joints as defined by swelling or effusion, or presence of 2 or more of the following:
Limitation of range of motion Tenderness or pain on motion Increased heat Duration of
disease 6 weeks or longer Onset type defined by type of disease in first 6 months:
Polyarthritis (i.e., 5 or more inflamed joints) Oligoarthritis (i.e., less than 5 inflamed
joints) Systemic (i.e., arthritis with characteristic fever) Exclusion of other forms of
juvenile arthritis Active disease evidenced by 1 of the following: Minimum of 2 swollen or
tender joints using the 71 joint count method Endocardial or myocardial disease, or
serositis Anemia or thrombocytosis of chronic disease High risk for developing deforming
joint disease or evidence of potential life threatening involvement for at least 1
internal organ system Radiographic evidence of erosive arthritis developing within first
24 months of clinical disease Functional class II or III Endocardial, myocardial,
pericardial, and/or pleural disease Hemoglobin less than 10.0 g/dL or platelet count
greater than 600,000/mm3 Refractory to conventional therapy after 12 months of
methotrexate used in combination with hydroxychloroquine, sulfasalazine, azathioprine,
cyclosporine, or cyclophosphamide OR Controlled on conventional therapy but at price of
unacceptable toxicity

Systemic lupus erythematosus: Malar rash Discoid rash Photosensitivity Oral ulcers
Arthritis Serositis Renal disorder Neurologic disorder Hematologic disorder Immunologic
disorder Antinuclear antibody Must have at least 4 of 7 variables on the lupus activity
scale measured Evidence of potential life threatening involvement of at least 1 internal
organ system Endocardial and/or myocardial disease Central nervous system disease
Pulmonary parenchymal disease Renal disease defined as WHO III, IV or V and a high
activity and low chronicity index Immune mediated cytopenias Refractory to conventional
therapy after attempts to control disease with at least 2 drugs, including prednisone and
1 of the following: Azathioprine Cyclophosphamide (greater than 500 mg/m2 monthly for 6
months) Cyclosporine OR Controlled on conventional therapy but at price of unacceptable
toxicity

Vasculitis Definitive diagnosis of 1 of the following forms: Churg-Strauss syndrome Giant
cell arteritis Henoch-Schonlein purpura Hypersensitivity vasculitis Polyarteritis nodosa
Takayasu arteritis Wegener's granulomatosis Evidence of active disease defined as
reversible manifestations of the underlying inflammatory process Must have 1 or more of
the following: Elevated Westergren erythrocyte sedimentation rate Elevated C reactive
protein Decrease serum complement levels Evidence of potential life threatening
involvement of at least 1 internal organ system Endocardial and/or myocardial disease
Central nervous system disease Pulmonary parenchymal disease Renal disease defined as WHO
III, IV or V and a high activity and low chronicity index Immune mediated cytopenias
Refractory to conventional therapy (i.e., failed or relapsed within 6 months) after
attempts to control disease with at least 2 drugs, including prednisone and 1 of the
following: Methotrexate Azathioprine Cyclophosphamide Cyclosporine OR Controlled on
conventional therapy but at price of unacceptable toxicity

Performance status: ECOG 0-1 ECOG 2 allowed provided symptoms directly related to
autoimmune disease Hepatic: No history of severe, prior or ongoing chronic liver disease
Bilirubin less than 2.0 mg/dL AST less than 2 times upper limit of normal (ULN) Alkaline
phosphatase less than 2 times ULN Renal: Creatini



Age minimum: 1 Year
Age maximum: 55 Years
Gender: Both
Health Condition(s) or Problem(s) studied
Anemia, Hemolytic, Autoimmune
Autoimmune Thrombocytopenic Purpura
Churg-Strauss Syndrome
Giant Cell Arteritis
Graft Versus Host Disease
Hypersensitivity Vasculitis
Juvenile Rheumatoid Arthritis
Polyarteritis Nodosa
Pure Red Cell Aplasia
Purpura, Schoenlein-Henoch
Rheumatoid Arthritis
Systemic Lupus Erythematosus
Takayasu Arteritis
Wegener's Granulomatosis
Intervention(s)
Drug: anti-thymocyte globulin
Drug: cyclophosphamide
Drug: cyclosporine
Drug: filgrastim
Drug: methylprednisolone
Drug: prednisone
Procedure: Autologous Peripheral Blood Stem Cell Transplantation
Primary Outcome(s)
Secondary Outcome(s)
Secondary ID(s)
199/15105
UMN-MT-1996-15
UMN-MT-9615
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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