Phase II Study of High-Dose Cyclophosphamide in Patients With Severe Autoimmune Hematologic Disease

Sponsors

Lead sponsor: Johns Hopkins University

Source Office of Rare Diseases (ORD)
Brief Summary

OBJECTIVES:

I. Determine the response rate and 1-year event-free survival in patients with severe autoimmune hematologic disease treated with high-dose cyclophosphamide.

Detailed Description

PROTOCOL OUTLINE: Patients receive high-dose cyclophosphamide IV on days 1-4 and filgrastim (G-CSF) starting on day 10 and continuing until blood counts recover.

Patients are followed at 1, 3, 6, and 12 months and then annually thereafter.

Overall Status Completed
Start Date March 1999
Primary Completion Date August 2007
Phase Phase 2
Study Type Interventional
Enrollment 32
Condition
Intervention

Intervention type: Drug

Intervention name: cyclophosphamide

Intervention type: Drug

Intervention name: filgrastim

Eligibility

Criteria:

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

- Diagnosis of severe autoimmune hematologic disease Autoimmune hemolytic anemia OR Immune thrombocytopenia

- Failure of at least 2 standard treatment approaches (e.g., prednisone therapy, splenectomy, intravenous immunoglobulin, or other immunosuppressants)

- Inability to taper prednisone dose to less than 10 mg/day OR Autoimmune neutropenia including the following: Felty's syndrome OR Disorders of large granular lymphocytes with recurrent infections or absolute neutrophil count less than 200/mm3

--Prior/Concurrent Therapy--

- See Disease Characteristics

--Patient Characteristics--

- Age: Not specified

- Performance status: Not specified

- Hematopoietic: See Disease Characteristics

- Hepatic: Not specified

- Renal: Creatinine no greater than 2.5 mg/dL

- Cardiovascular: Ejection fraction at least 40%

- Pulmonary: FVC, FEV1, or DLCO at least 50% predicted

Other:

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception

- Not preterminal or moribund

Gender: All

Minimum age: N/A

Maximum age: N/A

Healthy volunteers: No

Overall Official
Last Name Role Affiliation
Robert A. Brodsky Study Chair Johns Hopkins University
Location
facility Johns Hopkins Oncology Center
Location Countries

United States

Verification Date

September 2008

Keywords
Has Expanded Access No
Condition Browse
Study Design Info

Allocation: N/A

Intervention model: Single Group Assignment

Primary purpose: Treatment

Source: ClinicalTrials.gov