Sickle CellNCT00033774
Stem Cell Collection for Adult Volunteers
An observational study of Sickle Cell, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
This study will examine the development of stem cells (very immature cells produced by the bone marrow) and their potential to change into cells of other organ types. These cells will be studied for their potential use in creating replacement tissue for diseases ranging from diabetes to Parkinson s. Read more about this study
- Who is it for?
- Ages 18 to 100 yearsHealthy volunteers can take part
- Conditions the study is for
- Sickle Cell
- How many people
- 136 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Apr 2002to 19 Aug 2022
- 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 18 to 100 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Recruitment will occur from a pool of normal volunteers. Volunteers who are not undergoing pretreatment with G-CSF will receive two hundred and fifty (250) dollars for the leukapheresis. Volunteers who undergo pretreatment with G-CSF will receive a payment of five hundred (500) dollars upon completion of 5 days of G-CSF, blood draws, and the leukapheresis. In the event of early withdrawal from the study before the leukapheresis, the volunteer will receive fifty (50) dollars for each visit to the hospital which includes blood draws and G-CSF administration.
Who may be able to join
- Age 18 or greater.
- Normal renal function: creatinine less than1.5 mg/dL, proteinuria less than1+.
- Normal liver function: bilirubin less than 2.5 mg/dL, ALT less than 2.5 times the upper limit of normal, all other transminases less than 2.5 times the upper limit of normal.
- Normal blood counts: WBC 3,000-10,000/mm3, granulocytes greater than 1,500/mm3, platelets greater than 150,000/mm3, hemoglobin greater than 12.5g/dL, MCV within normal limits.
- Female volunteers of childbearing age should have a negative serum pregnancy test within one week of beginning G-CSF administration.
- Meets NIH Department of Transfusion Medicine (DTM) eligibility criteria for blood component donation for in vitro research use (negative serologic tests for syphilis, hepatitis B and C, HIV, and HTLV-1).
- Ability to give informed consent to participate in the protocol.
Who cannot join
- Any underlying hematologic disorder including sickle cell disease.
- Active viral, bacterial, fungal, or parasitic infection.
- History of autoimmune disease, such as rheumatoid arthritis or systemic lupus erythematosus.
- History of cancer excluding squamous carcinoma of the skin and cervical carcinoma in situ.
- History of cardiovascular disease or related symptoms such as chest pain or shortness of breath.
- Any positive serum screening test as listed below.
- Allergy to G-CSF or bacterial E. coli products.
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: Peripheral Blood Stem Cell Collection From Adult Volunteers
This study will examine the development of stem cells (very immature cells produced by the bone marrow) and their potential to change into cells of other organ types. These cells will be studied for their potential use in creating replacement tissue for diseases ranging from diabetes to Parkinson s.
Healthy volunteers 18 years of age or older may be eligible for this study. Candidates will be screened with a medical history, physical examination, and blood tests.
Participants will undergo a process called 'stem cell mobilization and apheresis' to collect bone marrow stem cells. For five days before the collection they will receive injections of a hormone called G-CSF, which stimulates release of stem cells from the bone marrow into the bloodstream. On the fifth day of the injections, stem cells will be collected through apheresis. For this procedure, blood is collected through a catheter (plastic tube) placed in an arm vein and directed into a cell separator machine. There, the white cells and stem cells are separated from the other blood components through a spinning process and collected in a bag inside the machine. The rest of the blood is returned to the donor through a catheter in the other arm.
Read the detailed description
The renewal of various tissues and organs at steady state or following damage relies upon a small population of locally residing tissue specific "stem cells" Stem cells from adult bone marrow represent an ideal stem cell source based on their ease of collection. In order to begin to explore the potential of adult bone marrow for the correction of genetic diseases that affect the blood such as sickle cell disease, we propose in vitro and in vivo mouse studies to examine the regulation of normal differentiation of hematopoietic stem cells collected from adult volunteers. In order to obtain adult hematopoietic stem cells in large numbers for in vitro and in vivo studies, volunteers will undergo mobilization with G-CSF for 5 consecutive days followed by large volume apheresis on the 5th day of G-CSF injection. The harvested product will be immunomagnetically purified for the primitive progenitor population and viably cryopreserved in multiple aliquots.
- Conditions
- Sickle Cell
- Keywords
- Leukapheresis, G-CSF, Mobilization, Stem Cells, Adult, Natural History, Healthy Volunteer, HV, Normal Control
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 |
|---|---|---|
| 1 | Eligible healthy volunteers 18 and older |
What is being measured
The primary outcome is the main question the study is designed to answer.
Collection of peripheral blood stem cells via standard leukapheresis
To explore the control of normal differentiation of adult hematopoietic stem cells collected from the peripheral blood after G-CSF mobilization.
Measured: 5 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2002 (estimated)
- Study start29 Apr 2002
- Primary completion27 Jun 2022
- Study completion19 Aug 2022
Why it stopped: There is another active NHLBI protocol for the collection of stem cells for research from healthy volunteers and no need for this separate Protocol.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Sickle CellStudies 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 136 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- John F Tisdale, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00033774
- Sponsor's protocol ID
- 020160
- Other IDs
- 02-H-0160
- Status last verified
- Aug 2022
- First submitted
- 9 Apr 2002
- Last updated
- 25 Aug 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Wei G, Schubiger G, Harder F, Muller AM. Stem cell plasticity in mammals and transdetermination in Drosophila: common themes? Stem Cells. 2000;18(6):409-14. doi: 10.1634/stemcells.18-6-409.
PubMed 11072028Orkin SH. Diversification of haematopoietic stem cells to specific lineages. Nat Rev Genet. 2000 Oct;1(1):57-64. doi: 10.1038/35049577.
PubMed 11262875Lemischka I. Stem cell dogmas in the genomics era. Rev Clin Exp Hematol. 2001 Mar;5(1):15-25. doi: 10.1046/j.1468-0734.2001.00030.x.
PubMed 11486729
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Peripheral Blood Stem Cell Collection From Adult Volunteers
- ClinicalTrials.gov ID
- NCT00033774
- Acronym / sponsor ID
- 020160
- Conditions
- Sickle Cell
- Keywords
- Leukapheresis, G-CSF, Mobilization, Stem Cells, Adult, Natural History, Healthy Volunteer, HV
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: There is another active NHLBI protocol for the collection of stem cells for research from healthy volunteers and no need for this separate Protocol.
- Enrolment
- 136 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 29 Apr 2002 (actual)
- Primary completion
- 27 Jun 2022 (actual)
- Study completion
- 19 Aug 2022 (actual)
- First posted
- 10 Apr 2002 (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 examine the development of stem cells (very immature cells produced by the bone marrow) and their potential to change into cells of other organ types. These cells will be studied for their potential use in creating replacement tissue for diseases ranging from diabetes to Parkinson s.
Healthy volunteers 18 years of age or older may be eligible for this study. Candidates will be screened with a medical history, physical examination, and blood tests.
Participants will undergo a process called 'stem cell mobilization and apheresis' to collect bone marrow stem cells. For five days before the collection they will receive injections of a hormone called G-CSF, which stimulates release of stem cells from the bone marrow into the bloodstream. On the fifth day of the injections, stem cells will be collected through apheresis. For this procedure, blood is collected through a catheter (plastic tube) placed in an arm vein and directed into a cell separator machine. There, the white cells and stem cells are separated from the other blood components through a spinning process and collected in a bag inside the machine. The rest of the blood is returned to the donor through a catheter in the other arm.
Detailed description
The renewal of various tissues and organs at steady state or following damage relies upon a small population of locally residing tissue specific "stem cells" Stem cells from adult bone marrow represent an ideal stem cell source based on their ease of collection. In order to begin to explore the potential of adult bone marrow for the correction of genetic diseases that affect the blood such as sickle cell disease, we propose in vitro and in vivo mouse studies to examine the regulation of normal differentiation of hematopoietic stem cells collected from adult volunteers. In order to obtain adult hematopoietic stem cells in large numbers for in vitro and in vivo studies, volunteers will undergo mobilization with G-CSF for 5 consecutive days followed by large volume apheresis on the 5th day of G-CSF injection. The harvested product will be immunomagnetically purified for the primitive progenitor population and viably cryopreserved in multiple aliquots.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | – | –Eligible healthy volunteers 18 and older |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Recruitment will occur from a pool of normal volunteers. Volunteers who are not undergoing pretreatment with G-CSF will receive two hundred and fifty (250) dollars for the leukapheresis. Volunteers who undergo pretreatment with G-CSF will receive a payment of five hundred (500) dollars upon completion of 5 days of G-CSF, blood draws, and the leukapheresis. In the event of early withdrawal from the study before the leukapheresis, the volunteer will receive fifty (50) dollars for each visit to the hospital which includes blood draws and G-CSF administration.
Key inclusion criteria (7 listed)
- Age 18 or greater.
- Normal renal function: creatinine less than1.5 mg/dL, proteinuria less than1+.
- Normal liver function: bilirubin less than 2.5 mg/dL, ALT less than 2.5 times the upper limit of normal, all other transminases less than 2.5 times the upper limit of normal.
- Normal blood counts: WBC 3,000-10,000/mm3, granulocytes greater than 1,500/mm3, platelets greater than 150,000/mm3, hemoglobin greater than 12.5g/dL, MCV within normal limits.
- Female volunteers of childbearing age should have a negative serum pregnancy test within one week of beginning G-CSF administration.
Key exclusion criteria (7 listed)
- Any underlying hematologic disorder including sickle cell disease.
- Active viral, bacterial, fungal, or parasitic infection.
- History of autoimmune disease, such as rheumatoid arthritis or systemic lupus erythematosus.
- History of cancer excluding squamous carcinoma of the skin and cervical carcinoma in situ.
- History of cardiovascular disease or related symptoms such as chest pain or shortness of breath.
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
Collection of peripheral blood stem cells via standard leukapheresis
Time frame: 5 days
To explore the control of normal differentiation of adult hematopoietic stem cells collected from the peripheral blood after G-CSF mobilization.
Results and safety
The study was stopped early (reason given: There is another active NHLBI protocol for the collection of stem cells for research from healthy volunteers and no need for this separate Protocol.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2022.
- Background: PubMed 11072028Wei G, Schubiger G, Harder F, Muller AM. Stem cell plasticity in mammals and transdetermination in Drosophila: common themes? Stem Cells. 2000;18(6):409-14. doi: 10.1634/stemcells.18-6-409.
- Background: PubMed 11262875Orkin SH. Diversification of haematopoietic stem cells to specific lineages. Nat Rev Genet. 2000 Oct;1(1):57-64. doi: 10.1038/35049577.
- Background: PubMed 11486729Lemischka I. Stem cell dogmas in the genomics era. Rev Clin Exp Hematol. 2001 Mar;5(1):15-25. doi: 10.1046/j.1468-0734.2001.00030.x.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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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.
John F Tisdale, 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 Sickle Cell, 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 7 inclusion and 7 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: stopped early, as of the sponsor's last update on 25 Aug 2022. 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 was stopped early (reason given: There is another active NHLBI protocol for the collection of stem cells for research from healthy volunteers and no need for this separate Protocol.). Results are not available in the source record.
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?
Collection of peripheral blood stem cells via standard leukapheresis (time frame: 5 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00033774, which also lists 3 linked publications.
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 25 Aug 2022.