Screening for Subjects to Participate in Studies of Blood Disorders



Status:Recruiting
Conditions:Anemia, Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any
Updated:12/2/2018
Start Date:June 3, 2008
Contact:Matthew M Hsieh, M.D.
Email:matthewhs@mail.nih.gov
Phone:(301) 402-7687

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Screening of Subjects to Determine Eligibility to Safely Participate in Blood Disorders Studies

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.

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.

- INCLUSION CRITERIA:

Subjects will be entered on this protocol at the time of their first visit to the NIH
Clinical Center outpatient clinic or inpatient service if the subject is able to give
consent/assent and the subject may have a blood disorder, may be a stem cell transplant
donor, or may be a healthy volunteer.

- The subject or the subject s guardian is capable of informed consent and willing to
sign the consent form after initial counseling by clinical staff. Additional consent
for clinically indicated procedures (central venous catheter placement; biopsy of
liver, kidney; or bone marrow) or blood transfusions will be obtained.

- The subject has a reasonable likelihood of having a disorder for which MMB or MCHB has
an active research protocol, and based on information received from an outside
physician, he/she appears to meet at least preliminary eligibility criteria for that
protocol.

- The subject has been identified as a potential donor for a subject for whom the MMB or
MCHB has an active stem cell transplant protocol and based on information received
from an outside physician, he/she appears to meet preliminary eligibility as a donor.

- The subject (age greater than or equal to18) is a healthy volunteer for whom the MMB
or MCHB has an active study recruiting healthy normal volunteers and he/she appears to
meet preliminary eligibility as a healthy volunteer.

EXCLUSION CRITERIA:

- Unable to comprehend the investigational nature of the protocol participation

- Abnormal CBC for healthy volunteers

- Positive pregnancy test for stem cell donors and healthy volunteers
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Phone: 800-411-1222
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Bethesda, MD
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