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

The recruitment status of this study is unknown because the information has not been verified recently.

Verified March 2007 by Office of Rare Diseases (ORD). Recruitment status was Active, not recruiting

First Received on October 18, 1999. Last Updated on March 28, 2007 History of Changes

Sponsor:	Johns Hopkins University
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00004464

Purpose

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:

Genetics Home Reference related topics: paroxysmal nocturnal hemoglobinuria

MedlinePlus related topics: Anemia Aplastic Anemia

Drug Information available for: Cyclophosphamide Filgrastim Lenograstim Granulocyte colony-stimulating factor

U.S. FDA Resources

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.

Eligibility

Ages Eligible for Study:	up to 70 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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, 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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004464

Locations

United States, Maryland
Johns Hopkins Oncology Center
Baltimore, Maryland, United States, 21231

Sponsors and Collaborators

Johns Hopkins University

Investigators

Study Chair:

Robert A. Brodsky

Johns Hopkins University

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00004464 History of Changes
Other Study ID Numbers:	199/13895, JHOC-96011702, JHOC-9611
Study First Received:	October 18, 1999
Last Updated:	March 28, 2007
Health Authority:	Unspecified

Keywords provided by Office of Rare Diseases (ORD):

aplastic anemia
hematologic disorders
paroxysmal nocturnal hemoglobinuria
rare disease

Additional relevant MeSH terms:

Anemia
Anemia, Aplastic
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Hematologic Diseases
Bone Marrow Diseases
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Myelodysplastic Syndromes
Cyclophosphamide

Lenograstim
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Adjuvants, Immunologic

ClinicalTrials.gov processed this record on February 09, 2012