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

This study has been completed.

First Received: February 2, 2001 Last Updated: September 8, 2008 History of Changes

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

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

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

Resource links provided by NLM:

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

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

No publications provided

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

Immunologic Factors
Arthritis, Rheumatoid
Cyclophosphamide
Hemostatic Disorders
Purpura, Thrombocytopenic
Signs and Symptoms
Thrombocytopenia
Musculoskeletal Diseases
Arthritis
Felty's Syndrome
Connective Tissue Diseases
Alkylating Agents
Purpura
Autoimmune Diseases
Hematologic Diseases

Joint Diseases
Blood Coagulation Disorders
Blood Platelet Disorders
Rare Diseases
Anemia
Anemia, Hemolytic
Rheumatic Diseases
Anemia, Hemolytic, Autoimmune
Immunosuppressive Agents
Autoimmune Hemolytic Anemia
Neutropenia
Thrombocytopathy
Antineoplastic Agents, Alkylating
Antirheumatic Agents

Additional relevant MeSH terms:

Skin Manifestations
Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Antineoplastic Agents
Physiological Effects of Drugs
Arthritis, Rheumatoid
Cyclophosphamide
Purpura, Thrombocytopenic
Signs and Symptoms
Pathologic Processes
Thrombocytopenia
Musculoskeletal Diseases
Therapeutic Uses
Arthritis
Syndrome

Felty's Syndrome
Connective Tissue Diseases
Alkylating Agents
Purpura
Autoimmune Diseases
Disease
Immune System Diseases
Hematologic Diseases
Joint Diseases
Blood Coagulation Disorders
Blood Platelet Disorders
Anemia
Anemia, Hemolytic
Rheumatic Diseases
Anemia, Hemolytic, Autoimmune

ClinicalTrials.gov processed this record on July 02, 2009