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Sponsors and Collaborators: |
Office of Rare Diseases (ORD) Rare Diseases Clinical Research Network |
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Information provided by: | Office of Rare Diseases (ORD) |
ClinicalTrials.gov Identifier: | NCT00315419 |
Bone marrow failure syndromes (BMFS) are rare disorders characterized by dysfunctional hematopoietic stem cells, which give rise to all red and white blood cells. The deficiency of blood cells, or cytopenia, caused by this malfunction leads to an assortment of diseases and disorders, all of which are characterized as BMFS. Because these diseases are rare, conducting research on them is difficult, and standards of treatment for most BMFS have yet to be developed. This study will collect clinical and laboratory data from people with BMFS to identify the characteristics and biological markers associated with these diseases over time. This information will assist doctors and researchers to develop better therapies and diagnostic tests that will help improve the management of BMFS and cytopenias.
Condition |
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Bone Marrow Failure Syndromes Anemia, Aplastic Myelodysplastic Syndromes Hemoglobinuria, Paroxysmal Red-Cell Aplasia, Pure Purpura, Thrombocytopenic Leukemia, Lymphocytic |
Study Type: | Observational |
Study Design: | Cohort, Prospective |
Official Title: | Screening Protocol and Longitudinal Study of Bone Marrow Failure Syndromes and Cytopenias |
Residual samples from peripheral blood and bone marrow aspiration/biopsies
Estimated Enrollment: | 450 |
Study Start Date: | April 2006 |
Estimated Study Completion Date: | July 2009 |
Estimated Primary Completion Date: | July 2009 (Final data collection date for primary outcome measure) |
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.
Ages Eligible for Study: | 11 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
Individuals with bone marrow failure syndromes
Inclusion Criteria:
Exclusion Criteria:
Contact: Susan Sunkle | 216-445-2119 | sunkles@ccf.org |
United States, California | |
University of California, Los Angeles, Department of Hematology and Oncology | Recruiting |
Los Angeles, California, United States, 90095 | |
Principal Investigator: Ronald Paquette, MD | |
United States, Florida | |
H. Lee Moffitt Cancer Center | Recruiting |
Tampa, Florida, United States, 33612 | |
Principal Investigator: Alan List, MD | |
Principal Investigator: P. K. Eppling-Burnette, PhD | |
United States, Ohio | |
Cleveland Clinic Foundation | Recruiting |
Cleveland, Ohio, United States, 44195 | |
Principal Investigator: Jaroslaw P. Maciejewski, MD, PhD | |
Principal Investigator: Mikkael Sekeres, MD | |
Principal Investigator: Anjali Advani, MD | |
Principal Investigator: Alan Lichtin, MD | |
Principal Investigator: Ronald Sobecks, MD | |
Principal Investigator: Matt Kalaycio, MD | |
Principal Investigator: Brad Pohlman, MD | |
Principal Investigator: Brian Bolwell, MD | |
United States, Pennsylvania | |
Pennsylvania State University Cancer Center | Active, not recruiting |
Hershey, Pennsylvania, United States, 17033 |
Study Chair: | Jaroslaw P. Maciejewski, MD, PhD | The Cleveland Clinic |
Responsible Party: | Cleveland Clinic Foundation ( Jaroslaw Maciejewski, MD, PhD ) |
Study ID Numbers: | RDCRN 5401, RR19397-03 |
Study First Received: | April 14, 2006 |
Last Updated: | September 2, 2008 |
ClinicalTrials.gov Identifier: | NCT00315419 |
Health Authority: | United States: Federal Government |
Large Granular Lymphocyte Leukemia Amegakaryocytic Thrombocytopenic Purpura Idiopathic Pure Red Cell Aplasia Paroxysmal Nocturnal Hemoglobinuria |
Paroxysmal nocturnal hemoglobinuria Leukemia, Lymphoid Precancerous Conditions Pure red cell aplasia Bone Marrow failure syndromes Hemostatic Disorders Purpura, Thrombocytopenic Red-Cell Aplasia, Pure Leukemia Signs and Symptoms Preleukemia Thrombocytopenia Urologic Diseases Hemoglobinuria, Paroxysmal Anemia, Aplastic |
Myelodysplastic syndromes Purpura Large granular lymphocyte leukemia Immunoproliferative Disorders Hematologic Diseases Urination Disorders Blood Platelet Disorders Blood Coagulation Disorders Myelodysplasia Myelodysplastic Syndromes Anemia Anemia, Hemolytic Pancytopenia Thrombocytopathy Hemoglobinuria |
Skin Manifestations Urological Manifestations Neoplasms Pathologic Processes |
Disease Neoplasms by Histologic Type Immune System Diseases Syndrome |