Clinical trial NCT00695123: Screening for Subjects to Participate in Studies of Blood Disorders
Official title: Screening of Subjects to Determine Eligibility to Safely Participate in Blood Disorders Studies
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Bone Marrow Transplant
Sickle Cell Disease
G-CSF
Keywords: Bone Marrow Transplant
Sickle Cell Disease
G-CSF
Apheresis
Lead sponsor: National Institutes of Health Clinical Center (CC)
Sponsor class: NIH
First posted: 2008-06-11
Enrollment: 816
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: 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.

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.

Eligibility criteria: * 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

Primary outcomes:
measure: Evaluate consented subjects with blood disorders
timeFrame: ongoing
Secondary outcomes:
Other outcomes:
Locations:
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center, 9000 Rockville Pike
state: Maryland
Study officials:
affiliation: National Heart, Lung, and Blood Institute (NHLBI)
name: Matthew M Hsieh, M.D.
role: PRINCIPAL_INVESTIGATOR
References: