Screening for Subjects to Participate in Studies of Blood Disorders

NCT00695123 | Completed

• 2 other identifiers: 08015608-H-0156
• Study Type: observational
• Target: 816
• Locations: 1 country
• Sites: 1

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.

Conditions

Bone Marrow Transplant; Sickle Cell Disease; G-CSF

Keywords

Bone Marrow Transplant; Sickle Cell Disease; G-CSF; Apheresis

Outcome Measures

Primary Outcomes (1)

• Evaluate consented subjects with blood disorders

  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

  ongoing

Study Arms (1)

Only 1 participant group (cohort)

The subject may have a blood disorder, may be a stem cell transplant donor, or may be a healthy volunteer.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI): lead

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Matthew M Hsieh, M.D.

  National Heart, Lung, and Blood Institute (NHLBI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 7, 2008
• First Posted: June 11, 2008
• Study Start: July 3, 2008
• Primary Completion: April 2, 2021
• Study Completion: April 2, 2021
• Last Updated: April 8, 2021

Record last verified: 2021-04

Locations

US (1)