Fanconi AnemiaNCT00271089
Blood Cell Collection for Future Use in Individuals With Fanconi Anemia
A study testing PBSC Collection, Bone Marrow Harvest and CliniMacs Cell Selection System in people with Fanconi Anemia, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the CD34+ cells, which are responsible for producing various types of blood cells. Individuals with FA may experience fatigue, bleeding, and increased infections. Read more about this study
- Who is it for?
- Ages 1 year to 35 years
- Conditions the study is for
- Fanconi Anemia
- What is being tested?
- PBSC Collection, Bone Marrow Harvest and CliniMacs Cell Selection System
- Type of treatment
- Medical device, Surgery or procedure
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2004to Oct 2007
- Phase
- Interventional
- 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 1 year to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of FA
- Normal bone marrow cytogenetics within 3 months of study entry
- Absolute neutrophil count (ANC) level greater than 750
- Hemoglobin level greater than 8 without transfusion
- Platelet level greater than 30,000 without transfusion
- Must weigh at least 7.5 kg
Who cannot join
- Myloid or lymphoid leukemia
- Cytogenic abnormalities
- HIV infected
- Neoplastic or non-neoplastic disease of any major organ system that would compromise the ability to withstand the collection procedure
- Uncontrolled infection
- Unable to tolerate general anesthesia
- Known adverse reaction to E. Coli
- Pregnant or breastfeeding
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: Collection of Hematopoietic Cells From Patients With Fanconi Anemia (FA) for Future Autologous Reinfusion and Research
Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the CD34+ cells, which are responsible for producing various types of blood cells. Individuals with FA may experience fatigue, bleeding, and increased infections. The purpose of this study is to collect and purify blood cells from individuals with FA and store them for future therapeutic use.
Read the detailed description
FA is a rare, inherited disease that is caused by a gene defect to the CD34+ cells. It primarily affects an individual's bone marrow, resulting in decreased production of blood cells. The lack of white blood cells affects an individual's ability to fight infections, the lack of platelets may result in bleeding, and the lack of red blood cells usually leads to anemia. FA is typically diagnosed in childhood, and there is a high fatality rate. This study will use two methods to collect, purify, and store participant's CD34+ cells for future use in case of severe bone marrow failure. The collected cells will also be used by researchers to better understand the causes of FA and to possibly develop new treatments.
This study will enroll individuals with FA. All participants will undergo a bone marrow biopsy within 3 months of study entry. Based on the results of this biopsy, participants will undergo either a bone marrow harvest procedure or a cytokine mobilized peripheral blood stem cell (PBSC) collection procedure. Prior to both procedures, medical history will be reviewed, blood will be drawn, liver and kidney function will be evaluated, and a physical examination will be performed. Participants who undergo the bone marrow harvest procedure will be admitted to the hospital, with a possible overnight stay for observation. The following day, participants will have a physical examination and blood draw for laboratory testing. A blood and/or platelet transfusion may be required following the procedure.
Participants who undergo the PBSC procedure will be required to receive injections of G-CSF, a protein found normally in the body, twice a day for 4 to 8 days prior to the procedure; G-CSF has been found to help increase the amount of CD34+ cells in the blood. Once the CD34+ level is within a certain range, the PBSC procedure will begin through an IV placed in the arm or a temporary collection catheter placed under the participants' collarbone. Blood cells will be collected, with some cells separated out and the remainder of the cells infused back into the participant. The length of this procedure will vary for each participant; it will take 3 to 6 hours a day, for 1 to 4 days. Participants may require blood and/or platelet transfusions prior to and during the procedure.
Following the bone marrow harvest and PBSC procedures, CD34+ cells will be isolated in a laboratory. The majority of the cells will be frozen and stored for future use by the participants. A small portion of the cells will be available for researchers to perform experimental research to better understand FA.
The study completion date listed in this record was obtained from the "End Date" entered in the Protocol Registration and Results System (PRS) record.
- Conditions
- Fanconi Anemia
- Being tested
- PBSC Collection, Bone Marrow Harvest, CliniMacs Cell Selection System
- Medical subject headings
- Fanconi Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PBSC CollectionProcedure or surgery
Bone Marrow HarvestProcedure or surgery
CliniMacs Cell Selection SystemDevice
What is being measured
The primary outcome is the main question the study is designed to answer.
CD34+ cell collection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2005 (estimated)
- Study startAug 2004
- Study completionOct 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fanconi AnemiaStudies 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- Children's Hospital Medical Center, Cincinnati
- Investigators
- Stella Davies, MBBS, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00271089
- Sponsor's protocol ID
- 378
- Other IDs
- CCHMCEH001, R01HL081499-01A1
- Status last verified
- Dec 2007
- First submitted
- 29 Dec 2005
- Last updated
- 12 Jul 2016
- Results posted
- No
Publications
Kelly PF, Radtke S, von Kalle C, Balcik B, Bohn K, Mueller R, Schuesler T, Haren M, Reeves L, Cancelas JA, Leemhuis T, Harris R, Auerbach AD, Smith FO, Davies SM, Williams DA. Stem cell collection and gene transfer in Fanconi anemia. Mol Ther. 2007 Jan;15(1):211-9. doi: 10.1038/sj.mt.6300033.
PubMed 17164793
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Collection of Hematopoietic Cells From Patients With Fanconi Anemia (FA) for Future Autologous Reinfusion and Research
- ClinicalTrials.gov ID
- NCT00271089
- Acronym / sponsor ID
- 378
- Conditions
- Fanconi Anemia
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Collaborators
- Children's Hospital Medical Center, Cincinnati
- Study start
- Aug 2004
- Study completion
- Oct 2007 (actual)
- First posted
- 30 Dec 2005 (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.
Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the CD34+ cells, which are responsible for producing various types of blood cells. Individuals with FA may experience fatigue, bleeding, and increased infections. The purpose of this study is to collect and purify blood cells from individuals with FA and store them for future therapeutic use.
Detailed description
FA is a rare, inherited disease that is caused by a gene defect to the CD34+ cells. It primarily affects an individual's bone marrow, resulting in decreased production of blood cells. The lack of white blood cells affects an individual's ability to fight infections, the lack of platelets may result in bleeding, and the lack of red blood cells usually leads to anemia. FA is typically diagnosed in childhood, and there is a high fatality rate. This study will use two methods to collect, purify, and store participant's CD34+ cells for future use in case of severe bone marrow failure. The collected cells will also be used by researchers to better understand the causes of FA and to possibly develop new treatments.
This study will enroll individuals with FA. All participants will undergo a bone marrow biopsy within 3 months of study entry. Based on the results of this biopsy, participants will undergo either a bone marrow harvest procedure or a cytokine mobilized peripheral blood stem cell (PBSC) collection procedure. Prior to both procedures, medical history will be reviewed, blood will be drawn, liver and kidney function will be evaluated, and a physical examination will be performed. Participants who undergo the bone marrow harvest procedure will be admitted to the hospital, with a possible overnight stay for observation. The following day, participants will have a physical examination and blood draw for laboratory testing. A blood and/or platelet transfusion may be required following the procedure.
Participants who undergo the PBSC procedure will be required to receive injections of G-CSF, a protein found normally in the body, twice a day for 4 to 8 days prior to the procedure; G-CSF has been found to help increase the amount of CD34+ cells in the blood. Once the CD34+ level is within a certain range, the PBSC procedure will begin through an IV placed in the arm or a temporary collection catheter placed under the participants' collarbone. Blood cells will be collected, with some cells separated out and the remainder of the cells infused back into the participant. The length of this procedure will vary for each participant; it will take 3 to 6 hours a day, for 1 to 4 days. Participants may require blood and/or platelet transfusions prior to and during the procedure.
Following the bone marrow harvest and PBSC procedures, CD34+ cells will be isolated in a laboratory. The majority of the cells will be frozen and stored for future use by the participants. A small portion of the cells will be available for researchers to perform experimental research to better understand FA.
The study completion date listed in this record was obtained from the "End Date" entered in the Protocol Registration and Results System (PRS) record.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 1 year to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Diagnosis of FA
- Normal bone marrow cytogenetics within 3 months of study entry
- Absolute neutrophil count (ANC) level greater than 750
- Hemoglobin level greater than 8 without transfusion
- Platelet level greater than 30,000 without transfusion
Key exclusion criteria (8 listed)
- Myloid or lymphoid leukemia
- Cytogenic abnormalities
- HIV infected
- Neoplastic or non-neoplastic disease of any major organ system that would compromise the ability to withstand the collection procedure
- Uncontrolled infection
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PBSC Collection | Procedure or surgery | – |
| Bone Marrow Harvest | Procedure or surgery | – |
| CliniMacs Cell Selection System | Device | – |
Primary outcome
CD34+ cell collection
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 12 Jul 2016.
- Background: PubMed 17164793Kelly PF, Radtke S, von Kalle C, Balcik B, Bohn K, Mueller R, Schuesler T, Haren M, Reeves L, Cancelas JA, Leemhuis T, Harris R, Auerbach AD, Smith FO, Davies SM, Williams DA. Stem cell collection and gene transfer in Fanconi anemia. Mol Ther. 2007 Jan;15(1):211-9. doi: 10.1038/sj.mt.6300033.
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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.
Stella Davies, MBBS principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PBSC Collection, Bone Marrow Harvest and CliniMacs Cell Selection System in people with Fanconi Anemia, 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 6 inclusion and 8 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 12 Jul 2016. 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?
CD34+ cell collection. 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 NCT00271089, which also lists 1 linked publication.
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 12 Jul 2016.