Screening for Subjects to Participate in Studies of Blood Disorders
- Conditions
- G-CSFSickle Cell DiseaseBone Marrow Transplant
- Registration Number
- NCT00695123
- Brief Summary
This study will determine eligibility for participation in research studies on blood disorders conducted by the National Heart, Lung and Blood Institute and the National Institute of Diabetes, Digestive, and Kidney Diseases.
Healthy volunteers, patients with blood disorders under study by NHLBI and NIDDK and potential stem cell donors for patients with blood disorders who are 8 years of age and older may be eligible for this screening protocol. (Healthy volunteers who qualify for research protocols would serve as control subjects.)
Participants undergo the following tests and procedures:
Healthy Volunteers
* Medical history, physical examination, blood tests and urine sample collection.
* Buccal mucosa sample collection. (Cells are collected from the inside of the cheek by gentle scraping with a bristly brush.)
* Bone marrow aspiration (only for volunteers 18 years of age and older).
Potential Stem Cell Donor
-Same as for healthy volunteers plus evaluations that may include electrocardiogram, echocardiogram, imaging studies (X-rays, CT scans, MRI scans and others), heart evaluation, and lung function tests.
Patient with Blood Disorder
* Same as for stem cell donors plus additional evaluations and treatments that may include radiation oncology evaluation, catheter placement, blood transfusions, kidney and liver biopsies. Short courses of drug treatment for induction of fetal hemoglobin in sickle cell patients, and/or iron chelation in patients receiving chronic red cell transfusions may be included as well.
- Detailed Description
This study allows the evaluation of subjects in order to determine their ability to safely participate in other active NIH research studies studying blood disorders. This protocol serves several purposes: 1) allows detailed investigations into the blood disorders of these subjects, and the status of other organ systems that would determine their ability to safely tolerate specific aspects of active research protocols; 2) allows investigators to offer clinical diagnostic testing and procedures to patients if this treatment will facilitate their participation in a clinical trial; 3) allows investigations as to whether a donor is HLA matched, fit to receive G-CSF, and fit to undergo apheresis and therefore eligible to participate as a donor on a bone marrow transplant protocol; 4) allows the evaluations of healthy volunteers as to whether they are eligible for participation as control subjects on protocols that require generally good health status by history or physical exam findings, or laboratory assessments; and 5) allows for the collection and storage of research specimens and samples obtainable by minimal risk for development or validation of novel clinical tools and approaches to disease. After completion of the screening process, the subject will either be offered a chance to participate in an active research protocol, or if no appropriate protocol is identified, subjects with blood disorders will have recommendations for other treatment options relayed to them and/or to the primary or referring physician.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 816
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Evaluate consented subjects with blood disorders ongoing 1) to assess the hematologic problems, the status of other organ systems, and their ability to safely tolerate active treatment protocols; 2) to determine eligibility for active MMB and/or MCHB protocols. Subjects may require screening procedures and clinical treatments in order to determine eligibility for participation; and 3) to allow time for counseling and deliberation regarding best treatment option
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
National Institutes of Health Clinical Center, 9000 Rockville Pike
🇺🇸Bethesda, Maryland, United States