Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

• Status: Completed
• Trial end date: 2008-11-11
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Johns Hopkins University; Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
• Treatments: Cyclophosphamide

Criteria

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

• 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