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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

69 participants

Study Classification

INTERVENTIONAL

Study Start Date

1996-02-29

Study Completion Date

2008-11-11

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Aplastic Anemia Paroxysmal Hemoglobinuria, Nocturnal

Study Design

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Primary Study Purpose

TREATMENT

Interventions

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cyclophosphamide

Intervention Type DRUG

filgrastim

Intervention Type DRUG

Eligibility Criteria

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Inclusion 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

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No