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

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ClinicalTrials.gov Identifier: NCT00004464
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : January 18, 2019
Sponsor:
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Brief Summary:

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 or disease Intervention/treatment Phase
Aplastic Anemia Paroxysmal Hemoglobinuria, Nocturnal Drug: cyclophosphamide Drug: filgrastim Phase 2

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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 69 participants
Primary Purpose: Treatment
Official Title: High Dose Cyclophosphamide for the Treatment of Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Study Start Date : February 1996
Actual Primary Completion Date : March 1, 2007
Actual Study Completion Date : November 11, 2008






Information from the National Library of Medicine

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Ages Eligible for Study:   up to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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
      • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004464


Locations
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United States, Maryland
Johns Hopkins Oncology Center
Baltimore, Maryland, United States, 21231
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Investigators
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Study Chair: Robert A. Brodsky Johns Hopkins University

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Responsible Party: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
ClinicalTrials.gov Identifier: NCT00004464     History of Changes
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 )
First Posted: October 19, 1999    Key Record Dates
Last Update Posted: January 18, 2019
Last Verified: August 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins:
aplastic anemia
hematologic disorders
paroxysmal nocturnal hemoglobinuria
rare disease

Additional relevant MeSH terms:
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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
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists