An observational study in Bone Marrow Diseases, sponsored by National Heart, Lung, and Blood Institute (NHLBI). Completed at 1 site in United States. Open to participants aged 2 Years to 80 Years. Per ClinicalTrials.gov, last updated 2020-09-10.
Sponsored by National Heart, Lung, and Blood Institute (NHLBI) · Observational
This study will examine whether cytokine gene polymorphisms affect the progression or response to therapy of bone marrow failure disorders. Cytokine genes instruct cells to produce proteins called cytokines that influence immune system response. As with many genes, the cytokine genes differ slightly from person to person. These differences are called gene polymorphisms. Different patients with the same bone marrow failure disease often progress and respond to treatment differently. This study will look at the possible role of cytokine gene polymorphisms in these differences.
Patients between 2 and 80 years old who have participated in an NHLBI Hematology Branch treatment protocol for acquired aplastic anemia, myelodysplastic syndrome, or pure red cell aplasia are recruited to participate in this study. Blood collected and stored at the time of screening for the treatment protocol will be tested for cytokine gene polymorphisms. No additional tests, procedures, or treatments are involved in this study.
The NHLBI Hematology Branch is investigating features that may affect the clinical course of bone marrow failure patients.
We are particularly interested in identifying factors, which determine treatment response and outcome. Cytokines are biological mediators of the immune response. In a normal population there is considerable variation in the precise sequence of the genes which control cytokine production (Cytokine Gene Polymorphism or CGP). As a consequence individuals differ in the quality of the immune response they mount against self or foreign antigens. Since the bone marrow failure disorders aplastic anemia and myelodysplastic syndrome involve auto-immune suppression of marrow function, it is important to discover whether there are any recurrent patterns of cytokine production in these disorders which may contribute to the marrow failure. This can be done by studying the sequences of the genes that control cytokine production to find out whether there are any recurrent gene patterns in the diseases studied. In addition we need to understand why some patients fail to respond to immunosuppressive treatments. By comparing CGP in responders and non-responders we may be able to find patterns of cytokine production that are favorable or unfavorable for response. Better understanding of CGPs in marrow failure syndromes should make it possible to improve the outcome for patients who fail immune suppression by using drugs which block specific cytokines.
None of these polymorphisms are associated with known clinical disease to be classifiable as a 'genetic defect'. All testing will be done on samples collected and stored for research purposes from consenting bone marrow failure subjects who have or will be participating on Hematology Branch research protocols.
66 studies on the registry are indexed under Bone Marrow Diseases; 12 are open to participants now.
This study's enrollment of 79 is below the median of 154 across 13 observational studies indexed under Bone Marrow Diseases.
Browse Bone Marrow Diseases studies →National Heart, Lung, and Blood Institute (NHLBI) is the lead sponsor of 1,143 studies on the registry; 71 are open to participants now.
Of its 58 completed or terminated interventional studies of FDA-regulated products, 51 (88%) have results posted.
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This is an ancillary study to a primary treatment protocol. Clinical data from the treatment protocol will be used in this protocol's analyses. All testing will be done on samples collected and stored for research purposes from consenting bone marrow failure subjects who have or will be participating on Hematology Branch bone marrow failure treatment research protocols.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Participation on a Hematology Branch bone marrow failure treatment protocol.
Diagnosis with one of the following bone marrow failure conditions:
Acquired aplastic anemia
Myelodysplastic syndrome (MDS)
Pure red cell aplasia (PRCA)
For adults: Ability to comprehend the nature of the study and provide . For minors: Written informed consent from one parent or guardian. Informed assent from minors: The process will be explained to the minor on a level of complexity appropriate for their age and ability to comprehend.
Age greater than or equal to 2 and less than or equal to 80.
EXCLUSION CRITERIA:
Subjects unable to comprehend the nature of the laboratory research.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bone marrow failure subjects
To define the variability that exists in cytokine genes from bone marrow failure patients by typing their DNA for polymorphisms.
Ongoing
Time frame: Ongoing
Compare cytokine polymorphisms of normal individuals (public domain studies and 03-H-0121) to those of patients with known bone marrow failure.Correlate cytokine gene polymorphisms of aplastic anemia and other bone marrow failure syndrome patien...
Time frame: Ongoing
Ran from Feb 2005 to primary completion in Mar 2007, 25 months.
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This study is completed, as verified in Sep 2020. You cannot join it, but the record below documents what was studied.
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National Heart, Lung, and Blood Institute (NHLBI) →