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

Purpose

OBJECTIVES:

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

Condition	Intervention	Phase
Anemia, Hemolytic, Autoimmune Felty Syndrome Purpura, Thrombocytopenic Autoimmune Diseases	Drug: cyclophosphamide Drug: filgrastim	Phase II

Genetics Home Reference related topics:

hemophilia thrombotic thrombocytopenic purpura

MedlinePlus related topics:

Anemia Autoimmune Diseases

Drug Information available for:

Cyclophosphamide Filgrastim

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Single Group Assignment

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment:	32
Study Start Date:	March 1999
Primary Completion Date:	August 2007 (Final data collection date for primary outcome measure)

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.

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00010387

Locations


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

Sponsors and Collaborators

Johns Hopkins University

Investigators


Study Chair:	Robert A. Brodsky	Johns Hopkins University

More Information

Study ID Numbers:	199/15672, JHOC-J9881, JHOC-99012906
First Received:	February 2, 2001
Last Updated:	September 8, 2008
ClinicalTrials.gov Identifier:	NCT00010387
Health Authority:	Unspecified

Keywords provided by Office of Rare Diseases (ORD):

Felty syndrome

autoimmune hemolytic anemia

autoimmune neutropenia

hematologic disorders

immune thrombocytopenic purpura

rare disease

Study placed in the following topic categories:

Purpura

Autoimmune hemolytic anemia

Autoimmune Diseases

Hematologic Diseases

Blood Platelet Disorders

Blood Coagulation Disorders

Joint Diseases

Anemia

Rare Diseases

Arthritis, Rheumatoid

Anemia, Hemolytic

Cyclophosphamide

Rheumatic Diseases

Hemostatic Disorders

Anemia, Hemolytic, Autoimmune

Purpura, Thrombocytopenic

Thrombocytopathy

Signs and Symptoms

Neutropenia

Thrombocytopenia

Musculoskeletal Diseases

Arthritis

Felty's Syndrome

Connective Tissue Diseases

Additional relevant MeSH terms:

Skin Manifestations

Disease

Molecular Mechanisms of Pharmacological Action

Immune System Diseases

Immunologic Factors

Antineoplastic Agents

Physiological Effects of Drugs

Immunosuppressive Agents

Pharmacologic Actions

Pathologic Processes

Syndrome

Therapeutic Uses

Myeloablative Agonists

Antineoplastic Agents, Alkylating

Antirheumatic Agents

Alkylating Agents

ClinicalTrials.gov processed this record on November 20, 2008

Sponsored by:	Johns Hopkins University
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00010387