Identifying Characteristics of Bone Marrow Failure Syndromes

Status unconfirmed

Conditions studied: Bone Marrow Failure Syndromes, Anemia, Aplastic, Myelodysplastic Syndromes, Hemoglobinuria, Paroxysmal, Red-Cell Aplasia, Pure, Purpura, Thrombocytopenic, Leukemia, Lymphocytic

In brief

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.

Key facts

Study ID

NCT00315419

Run by

Office of Rare Diseases (ORD)

People needed

450

Starts

2006-04-01

Expected to finish

2009-07-01

Last updated by the study team

2010-04-21

Who can join

Age: 11 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of one of the following diseases: aplastic anemia; myelodysplastic syndrome; paroxysmal nocturnal hemoglobinuria; idiopathic pure red cell aplasia; amegakaryocytic thrombocytopenia purpura; or large granular lymphocyte leukemia

You may not qualify if…

• N/A

Where it is running

• University of California, Los Angeles, Department of Hematology and Oncology — Los Angeles, California, United States
• H. Lee Moffitt Cancer Center — Tampa, Florida, United States
• Cleveland Clinic Foundation — Cleveland, Ohio, United States
• Pennsylvania State University Cancer Center — Hershey, Pennsylvania, United States

Full record on ClinicalTrials.gov

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