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

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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Key information

Conditions

Aplastic Anemia Anemia Anemia, Aplastic Anemia, Hemolytic Bone Marrow Diseases Bone Marrow Failure Disorders Disease Attributes Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes Paroxysmal Hemoglobinuria, Nocturnal Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases

Age range

Up to 70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Johns Hopkins Oncology Center

Baltimore, Maryland, 21231, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Cyclophosphamide

Drug

Filgrastim

Drug

Sponsors and collaborators

Lead sponsor

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Other

Registry information

Official study title

High Dose Cyclophosphamide for the Treatment of Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Important dates

Study start: 1996
Primary completion: 2007
Study completion: 2008
First posted: Oct 19, 1999
Registry last updated: Jan 18, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed