Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
This trial is active, not recruiting.
|Conditions||aplastic anemia, paroxysmal hemoglobinuria, nocturnal|
|Sponsor||Johns Hopkins University|
|Start date||February 1996|
|Trial size||25 participants|
|Trial identifier||NCT00004464, 199/13895, JHOC-96011702, JHOC-9611|
OBJECTIVES: I. Confirm the efficacy demonstrated in a pilot study using high dose cyclophosphamide in patients with severe aplastic anemia.
II. Determine whether the addition of filgrastim (G-CSF) to high dose cyclophosphamide shortens the time to recovery in these patients.
III. Determine whether this regimen is efficacious in treating paroxysmal nocturnal hemoglobinuria.
Male or female participants up to 70 years old.
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Acquired severe aplastic anemia or paroxysmal nocturnal hemoglobinuria Not a candidate for allogeneic bone marrow transplantation Must meet one of the following criteria: - Severe aplastic anemia Less than 25% bone marrow cellularity and depression in two of three blood counts (reticulocytes less than 40,000/mm3, platelet count less than 20,000/mm3 and granulocytes less than 500/mm3) - Life-threatening paroxysmal nocturnal hemoglobinuria Absolute neutrophil count less than 500/mm3, platelet transfusion dependent, or thrombotic disease No Fanconi anemia No abnormal cytogenetics --Patient Characteristics-- Renal: Creatinine no greater than 2.0 mg/dL Cardiovascular: Cardiac ejection fraction at least 45% Other: Not preterminal or moribund Not pregnant
|Description||PROTOCOL OUTLINE: Patients receive high dose cyclophosphamide IV on days 1-4. Beginning on day 10, patients receive filgrastim (G-CSF) until the absolute neutrophil count is greater than 1,000/mm3 for 2 consecutive days. Patients are followed every 3 months for at least 2 years and annually thereafter.|
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