We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov Menu

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

This study has been completed.
ClinicalTrials.gov Identifier:
First Posted: February 2, 2001
Last Update Posted: September 10, 2008
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
Office of Rare Diseases (ORD)


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 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Primary Purpose: Treatment

Resource links provided by NLM:

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.


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--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


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • Not preterminal or moribund
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00010387

United States, Maryland
Johns Hopkins Oncology Center
Baltimore, Maryland, United States, 21231
Sponsors and Collaborators
Johns Hopkins University
Study Chair: Robert A. Brodsky Johns Hopkins University
  More Information

ClinicalTrials.gov Identifier: NCT00010387     History of Changes
Other Study ID Numbers: 199/15672
First Submitted: February 2, 2001
First Posted: February 2, 2001
Last Update Posted: September 10, 2008
Last Verified: September 2008

Keywords provided by Office of Rare Diseases (ORD):
Felty syndrome
autoimmune hemolytic anemia
autoimmune neutropenia
hematologic disorders
immune thrombocytopenic purpura
rare disease

Additional relevant MeSH terms:
Autoimmune Diseases
Purpura, Thrombocytopenic
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Felty Syndrome
Blood Coagulation Disorders
Hematologic Diseases
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Immune System Diseases
Thrombotic Microangiopathies
Blood Platelet Disorders
Arthritis, Rheumatoid
Joint Diseases
Musculoskeletal Diseases
Rheumatic Diseases
Connective Tissue Diseases
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating