Identifying Characteristics of Bone Marrow Failure Syndromes

The recruitment status of this study is unknown because the information has not been verified recently.
Verified July 2009 by Office of Rare Diseases (ORD).
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
Rare Diseases Clinical Research Network
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00315419
First received: April 14, 2006
Last updated: April 19, 2010
Last verified: July 2009

April 14, 2006
April 19, 2010
April 2006
July 2009   (final data collection date for primary outcome measure)
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Not Provided
Complete list of historical versions of study NCT00315419 on ClinicalTrials.gov Archive Site
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Identifying Characteristics of Bone Marrow Failure Syndromes
Screening Protocol and Longitudinal Study of Bone Marrow Failure Syndromes and Cytopenias

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.

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.

Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Retention:   Samples With DNA
Description:

Residual samples from peripheral blood and bone marrow aspiration/biopsies

Non-Probability Sample

Individuals with bone marrow failure syndromes

  • Bone Marrow Failure Syndromes
  • Anemia, Aplastic
  • Myelodysplastic Syndromes
  • Hemoglobinuria, Paroxysmal
  • Red-Cell Aplasia, Pure
  • Purpura, Thrombocytopenic
  • Leukemia, Lymphocytic
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
450
July 2009
July 2009   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • N/A
Both
11 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00315419
RDCRN 5401, RR19397-03
Not Provided
Jaroslaw Maciejewski, MD, PhD, Cleveland Clinic Foundation
Office of Rare Diseases (ORD)
Rare Diseases Clinical Research Network
Study Chair: Jaroslaw P. Maciejewski, MD, PhD The Cleveland Clinic
Office of Rare Diseases (ORD)
July 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP