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

January 16, 2019 updated by: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

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

Completed

Conditions

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

Interventional

Enrollment (Actual)

69

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 70 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Investigators

  • Study Chair: Robert A. Brodsky, Johns Hopkins University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 1996

Primary Completion (Actual)

March 1, 2007

Study Completion (Actual)

November 11, 2008

Study Registration Dates

First Submitted

October 18, 1999

First Submitted That Met QC Criteria

October 18, 1999

First Posted (Estimate)

October 19, 1999

Study Record Updates

Last Update Posted (Actual)

January 18, 2019

Last Update Submitted That Met QC Criteria

January 16, 2019

Last Verified

August 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

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

Yes

Studies a U.S. FDA-regulated device product

No

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.

Clinical Trials on Aplastic Anemia

Clinical Trials on cyclophosphamide

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Ukraine

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe