Identifying Characteristics of Bone Marrow Failure Syndromes
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|ClinicalTrials.gov Identifier: NCT00315419|
Recruitment Status : Unknown
Verified July 2009 by Office of Rare Diseases (ORD).
Recruitment status was: Active, not recruiting
First Posted : April 18, 2006
Last Update Posted : April 21, 2010
|Condition or disease|
|Bone Marrow Failure Syndromes Anemia, Aplastic Myelodysplastic Syndromes Hemoglobinuria, Paroxysmal Red-Cell Aplasia, Pure Purpura, Thrombocytopenic Leukemia, Lymphocytic|
BMFS result from hematopoietic progenitor or stem cell failure within the bone marrow. Specific causes of this problem, however, have been difficult to identify, as BMFS occur sporadically. For the same reason, few studies have been conducted to find out more about these diseases and to develop more appropriate and effective therapies. Aplastic anemia (AA) is the most common of all BMFS. Other types of BMFS include the following: myelodysplastic syndrome (MDS); paroxysmal nocturnal hemoglobinuria (PNH); pure red cell aplasia (PRCA); amegakaryocytic thrombocytopenic purpura (ATP); and large granular lymphocyte leukemia (LGL leukemia). Though AA is the most common of the BMFS, all BMFS are closely related in terms of their symptoms and characteristics. This study will collect clinical and laboratory data from people with BMFS to identify the characteristics and biological markers specific to each disease as it evolves. This information will assist doctors and researchers to devise better therapies and diagnostic tests that will help improve the management of BMFS and cytopenias.
Participants in this observational study will report to the study site for an initial screening visit, followed by study visits every 6 months for at least 5 years. At each visit, participants will be interviewed and examined by a physician. Laboratory tests, including blood collection and a bone marrow aspirate, will also be performed. Data collected for this study's database will be used to determine the prevalence of clinical events and laboratory abnormalities over the course of disease, to study the evolution of disease parameters and symptoms, and to evaluate current therapies and diagnostic tests.
|Study Type :||Observational|
|Estimated Enrollment :||450 participants|
|Official Title:||Screening Protocol and Longitudinal Study of Bone Marrow Failure Syndromes and Cytopenias|
|Study Start Date :||April 2006|
|Estimated Primary Completion Date :||July 2009|
|Estimated Study Completion Date :||July 2009|
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): NCT00315419
|United States, California|
|University of California, Los Angeles, Department of Hematology and Oncology|
|Los Angeles, California, United States, 90095|
|United States, Florida|
|H. Lee Moffitt Cancer Center|
|Tampa, Florida, United States, 33612|
|United States, Ohio|
|Cleveland Clinic Foundation|
|Cleveland, Ohio, United States, 44195|
|United States, Pennsylvania|
|Pennsylvania State University Cancer Center|
|Hershey, Pennsylvania, United States, 17033|
|Study Chair:||Jaroslaw P. Maciejewski, MD, PhD||The Cleveland Clinic|