Bone Marrow TransplantNCT00695123
Screening for Subjects to Participate in Studies of Blood Disorders
An observational study of Bone Marrow Transplant and Sickle Cell Disease, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Bone Marrow Transplant, Sickle Cell Disease, G-CSF
- How many people
- 816 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Jul 2008to 2 Apr 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: 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.)
Read more
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.
Read the 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.
- Conditions
- Bone Marrow Transplant, Sickle Cell Disease, G-CSF
- Keywords
- Bone Marrow Transplant, Sickle Cell Disease, G-CSF, Apheresis
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate consented subjects with blood disorders
- 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
Measured: ongoing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2008 (estimated)
- Study start3 Jul 2008
- Primary completion2 Apr 2021
- Study completion2 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bone Marrow TransplantStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 816 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Matthew M Hsieh, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00695123
- Sponsor's protocol ID
- 080156
- Other IDs
- 08-H-0156
- Status last verified
- Apr 2021
- First submitted
- 7 Jun 2008
- Last updated
- 8 Apr 2021
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Screening of Subjects to Determine Eligibility to Safely Participate in Blood Disorders Studies
- ClinicalTrials.gov ID
- NCT00695123
- Acronym / sponsor ID
- 080156
- Conditions
- Bone Marrow Transplant, Sickle Cell Disease, G-CSF
- Keywords
- Bone Marrow Transplant, Sickle Cell Disease, G-CSF, Apheresis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 816 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 3 Jul 2008 (actual)
- Primary completion
- 2 Apr 2021 (actual)
- Study completion
- 2 Apr 2021 (actual)
- First posted
- 11 Jun 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Evaluate consented subjects with blood disorders
Time frame: 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2021.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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RecruitingObservational study
Natural History Study to Determine Drug Metabolism Phenotype and Appropriate Germline Source DNA in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant
Leukemia, Lymphoma
RecruitingObservational study
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RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Matthew M Hsieh, M.D. principal investigator
National Heart, Lung, and Blood Institute (NHLBI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Bone Marrow Transplant and Sickle Cell Disease, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate consented subjects with blood disorders (time frame: ongoing). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2021.