Identifying Characteristics of Bone Marrow Failure Syndromes

NCT00315419 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 450

Last updated 2010-04-21

No results posted yet for this study

Summary

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.

Conditions

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

Sponsors & Collaborators

  • Rare Diseases Clinical Research Network

    collaborator NETWORK
  • Office of Rare Diseases (ORD)

    lead NIH

Principal Investigators

  • Jaroslaw P. Maciejewski, MD, PhD · The Cleveland Clinic

Eligibility

Min Age
11 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2006-04-30
Primary Completion
2009-07-31
Completion
2009-07-31

Countries

  • United States

Study Locations

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Entities

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00315419 on ClinicalTrials.gov