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

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified March 2007 by Office of Rare Diseases (ORD).
Recruitment status was:  Active, not recruiting
Information provided by:
Office of Rare Diseases (ORD) Identifier:
First received: October 18, 1999
Last updated: March 28, 2007
Last verified: March 2007

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.

Condition Intervention
Aplastic Anemia
Paroxysmal Hemoglobinuria, Nocturnal
Drug: cyclophosphamide
Drug: filgrastim

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment: 25
Study Start Date: February 1996
Detailed 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.


Ages Eligible for Study:   up to 70 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--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

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Please refer to this study by its identifier: NCT00004464

United States, Maryland
Johns Hopkins Oncology Center
Baltimore, Maryland, United States, 21231
Sponsors and Collaborators
Johns Hopkins University
Study Chair: Robert A. Brodsky Johns Hopkins University
  More Information Identifier: NCT00004464     History of Changes
Other Study ID Numbers: 199/13895
Study First Received: October 18, 1999
Last Updated: March 28, 2007

Keywords provided by Office of Rare Diseases (ORD):
aplastic anemia
hematologic disorders
paroxysmal nocturnal hemoglobinuria
rare disease

Additional relevant MeSH terms:
Anemia, Aplastic
Hemoglobinuria, Paroxysmal
Hematologic Diseases
Bone Marrow Diseases
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Myelodysplastic Syndromes
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists processed this record on April 21, 2017