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

Aplastic Anemia Clinical Trial

Official title:

High Dose Cyclophosphamide for the Treatment of Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004464
• Other study ID #: J9611
• Secondary ID: 96-01-17-02JHOC-
• Status: Completed
• Phase: Phase 2
• Start date: February 1996
• Est. completion date: November 11, 2008

Study information

• Verified date: August 2018
• Source: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 69
• Est. completion date: November 11, 2008
• Est. primary completion date: March 1, 2007
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia
• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Hemoglobinuria, Nocturnal

Intervention

Drug:
• cyclophosphamide

• filgrastim

Locations

Country	Name	City	State
United States	Johns Hopkins Oncology Center	Baltimore	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Country where clinical trial is conducted

United States,