- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00004464
Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
High Dose Cyclophosphamide for the Treatment of 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.
Study Overview
Status
Intervention / Treatment
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.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Maryland
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Baltimore, Maryland, United States, 21231
- Johns Hopkins Oncology Center
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
Collaborators and Investigators
Investigators
- Study Chair: Robert A. Brodsky, Johns Hopkins University
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Urologic Diseases
- Urological Manifestations
- Bone Marrow Diseases
- Hematologic Diseases
- Urination Disorders
- Proteinuria
- Anemia, Hemolytic
- Bone Marrow Failure Disorders
- Myelodysplastic Syndromes
- Anemia
- Anemia, Aplastic
- Hemoglobinuria
- Hemoglobinuria, Paroxysmal
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Antirheumatic Agents
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Antineoplastic Agents, Alkylating
- Alkylating Agents
- Myeloablative Agonists
- Cyclophosphamide
Other Study ID Numbers
- J9611
- 96-01-17-02 (Other Identifier: JHM IRB)
- JHOC-96011702
- JHOC-9611 (Other Identifier: other)
- 199/13895 (Other Identifier: other)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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