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

Primary Purpose

Aplastic Anemia, Paroxysmal Hemoglobinuria, Nocturnal

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
cyclophosphamide
filgrastim
Sponsored by
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Aplastic Anemia focused on measuring aplastic anemia, hematologic disorders, paroxysmal nocturnal hemoglobinuria, rare disease

Eligibility Criteria

undefined - 70 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Johns Hopkins Oncology Center

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
October 18, 1999
Last Updated
January 16, 2019
Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
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1. Study Identification

Unique Protocol Identification Number
NCT00004464
Brief Title
Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Official Title
High Dose Cyclophosphamide for the Treatment of Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Study Type
Interventional

2. Study Status

Record Verification Date
August 2018
Overall Recruitment Status
Completed
Study Start Date
February 1996 (undefined)
Primary Completion Date
March 1, 2007 (Actual)
Study Completion Date
November 11, 2008 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

4. Oversight

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

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Aplastic Anemia, Paroxysmal Hemoglobinuria, Nocturnal
Keywords
aplastic anemia, hematologic disorders, paroxysmal nocturnal hemoglobinuria, rare disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Enrollment
69 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
cyclophosphamide
Intervention Type
Drug
Intervention Name(s)
filgrastim

10. Eligibility

Sex
All
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robert A. Brodsky
Organizational Affiliation
Johns Hopkins University
Official's Role
Study Chair
Facility Information:
Facility Name
Johns Hopkins Oncology Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21231
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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