Bone Marrow Failure SyndromePhase 1NCT07585136
Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders
A Phase 1 study testing Filgrastim, Plerixafor and Leukapheresis in people with Bone Marrow Failure Syndrome, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
The purpose of this study is to investigate mobilization and collection of HSPCs in patients with bone marrow failure syndromes (BMFS) using granulocyte-colony stimulating factor (otherwise known as Filgrastim) with plerixafor to demonstrate safety and feasibility of collecting HSPCs to advance gene therapy. Read more about this study
- Who is it for?
- Ages 18 to 25 years
- Conditions the study is for
- Bone Marrow Failure Syndrome
- What is being tested?
- Filgrastim, Plerixafor and Leukapheresis
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2026to 1 Jul 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 25 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Participants with a bone marrow failure syndrome with an identified genetic cause willing to donate autologous HSPCs for advancing gene therapy
- Age ≥ 18 years - 25 years
- The following hematological parameters need to be met (regardless of transfusion or growth factor support)
- Hb > 8 g/dL
- ANC > 500/mm3
- Platelet > 30,000/mm3
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- Bone marrow evaluation within the preceding 6 months prior to mobilization and apheresis
- Participants should either have a central venous catheter (CVC) in place, be able to undergo apheresis without requiring a CVC, or agree to having a temporary apheresis catheter placed
- Karnofsky score >80
- Negative serologic tests for syphilis, hepatitis B and C, HIV, and HTLV-1/II
- Female participants of childbearing age should have a negative serum pregnancy test within one week of beginning Filgrastim and plerixafor administration
Who cannot join
- Participant with sickle cell disease
- Participant who has had a prior autologous or allogeneic HSCT
- Active viral, bacterial, fungal, or parasitic infection
- Total bilirubin >2.5x ULN or transaminases >5x ULN
- Moderate or severe renal failure defined as serum/plasma creatinine >1.5 mg/dL and an estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 based on the CKD-Epi equation or the St. Jude equation
- Diagnosis of MDS or other hematologic malignancy
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- History of malignancy
- Known allergy to or contraindication for Filgrastim or plerixafor administration, or medications routinely administered during apheresis
- Splenomegaly (size greater than upper limit of normal on examination)
- Any disease or concomitant process that is not compatible with the study as per investigator opinion
- Concomitant treatment with alternative investigational agent or participation in another clinical trial with an investigational drug within 5 half-lives of the investigational agent
- Unwillingness to use a highly effective method of contraception for 1 month after plerixafor or GCSF
- Pregnancy
- Inability or unwillingness of research participant to give written informed consent.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105-2794
Alexis Leonard, MD, 888-226-4343, referralinfo@stjude.org
About this study
The purpose of this study is to investigate mobilization and collection of HSPCs in patients with bone marrow failure syndromes (BMFS) using granulocyte-colony stimulating factor (otherwise known as Filgrastim) with plerixafor to demonstrate safety and feasibility of collecting HSPCs to advance gene therapy.
Primary objective:
Read more
- To characterize the safety of Filgrastim plus plerixafor in participants with life threatening blood disorders as determined by the incidence of adverse events (AEs).
Secondary Objectives:
- To measure the mobilization effects of Filgrastim plus plerixafor in the peripheral blood in participants as determined by peak peripheral blood CD34+ counts.
- To characterize the feasibility of HSPC mobilization using Filgrastim plus plerixafor as determined by peripheral blood CD34+ counts.
- To estimate efficacy of Filgrastim plus plerixafor for HSPC mobilization and apheresis collection in participants as determined by the yield of CD34+ cells (CD34+ cells/kg).
Read the detailed description
This is a phase I, open-label, single-center study to evaluate the safety of Filgrastim plus plerixafor stem cell mobilization and apheresis in patients with BMFS. This study will include a screening period with labs, physical examination, and bone marrow evaluation at least 6 months prior to mobilization and apheresis, an intervention period that includes mobilization and apheresis of patient HSPCs, and outpatient follow-up within 7-10 days after intervention. Study staff will follow up with the participant via telephone approximately 30 days after mobilization and apheresis. A bone marrow evaluation will be done within 6 months post-intervention.
- Conditions
- Bone Marrow Failure Syndrome
- Being tested
- Filgrastim, Plerixafor, Leukapheresis
- Medical subject headings
- Bone Marrow Failure Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FilgrastimDrug
Administered twice daily dose starting on day 1 for 5 days.
PlerixaforDrug
Administered on day 5 via IV.
LeukapheresisProcedure or surgery
Peripheral venous access or through a central venous catheter approximately 4-5 hours after the dose of plerixafor is given.
| Study group | Role | What they receive |
|---|---|---|
| BDSTEM Treatment | Experimental | Participants in this study will receive a twice daily dose of Filgrastim (GCSF) (5 mcg/kg BID) SQ starting on day 1 for 5 days followed by a single dose of SQ plerixafor (0.24 mg/kg) on day 5 followed by collection of CD34+ HSPCs via apheresis.
A portion of cells collected from the participant will be stored as backup to be used toward future gene therapy endeavors. The remaining cells will be donated for research studies Receives: Drug: Filgrastim; Drug: Plerixafor; Procedure: Leukapheresis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events following filgrastim plus plerixafor administration
Safety will be assessed by the incidence, type, and severity of adverse events occurring after administration of filgrastim plus plerixafor in participants with bone marrow failure syndromes.
Measured: From initiation of drug administration through Day +7 to +10 follow-up
Secondary outcomes (4)
Number of participants achieving peripheral blood CD34+ counts ≥5 cells/µL
Feasibility of hematopoietic stem and progenitor cell mobilization will be assessed by peripheral blood CD34+ cell counts measured after plerixafor administration and prior to or during apheresis.
Measured: From initiation of plerixafor administration through completion of apheresis, or 6 hours after drug administration if apheresis is not performed
Peripheral blood CD34+ kinetics following filgrastim plus plerixafor administration
Peripheral blood CD34+ cell counts will be measured after plerixafor administration and prior to or during apheresis.
Measured: After plerixafor administration through completion of apheresis, or 6 hours after drug administration if apheresis is not performed
Observed CD34+ cell yield after 1 blood volume apheresis
CD34+ cell yield (CD34+ cells/kg) collected after processing 1 blood volume during apheresis following filgrastim plus plerixafor administration.
Measured: At completion of 1 blood volume apheresis on Day 5
Estimated total CD34+ cell yield from projected full-volume apheresis
Estimated total CD34+ cell yield (CD34+ cells/kg) projected from the observed yield after processing 1 blood volume during apheresis.
Measured: At completion of 1 blood volume apheresis on Day 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2026
- Study startNov 2026 (estimated)
- Primary completion1 Dec 2028 (estimated)
- Study completion1 Jul 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bone Marrow Failure SyndromeStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe 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 1
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Alexis Leonard, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07585136
- Sponsor's protocol ID
- BDSTEM
- Status last verified
- Oct 2026
- First submitted
- 16 Apr 2026
- Last updated
- 6 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders
- ClinicalTrials.gov ID
- NCT07585136
- Acronym / sponsor ID
- BDSTEM
- Conditions
- Bone Marrow Failure Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- Nov 2026 (estimated)
- Primary completion
- 1 Dec 2028 (estimated)
- Study completion
- 1 Jul 2029 (estimated)
- First posted
- 13 May 2026 (actual)
- 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.
The purpose of this study is to investigate mobilization and collection of HSPCs in patients with bone marrow failure syndromes (BMFS) using granulocyte-colony stimulating factor (otherwise known as Filgrastim) with plerixafor to demonstrate safety and feasibility of collecting HSPCs to advance gene therapy.
Primary objective:
- To characterize the safety of Filgrastim plus plerixafor in participants with life threatening blood disorders as determined by the incidence of adverse events (AEs).
Secondary Objectives:
- To measure the mobilization effects of Filgrastim plus plerixafor in the peripheral blood in participants as determined by peak peripheral blood CD34+ counts.
- To characterize the feasibility of HSPC mobilization using Filgrastim plus plerixafor as determined by peripheral blood CD34+ counts.
- To estimate efficacy of Filgrastim plus plerixafor for HSPC mobilization and apheresis collection in participants as determined by the yield of CD34+ cells (CD34+ cells/kg).
Detailed description
This is a phase I, open-label, single-center study to evaluate the safety of Filgrastim plus plerixafor stem cell mobilization and apheresis in patients with BMFS. This study will include a screening period with labs, physical examination, and bone marrow evaluation at least 6 months prior to mobilization and apheresis, an intervention period that includes mobilization and apheresis of patient HSPCs, and outpatient follow-up within 7-10 days after intervention. Study staff will follow up with the participant via telephone approximately 30 days after mobilization and apheresis. A bone marrow evaluation will be done within 6 months post-intervention.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BDSTEM Treatment | Experimental | Drug: Filgrastim, Drug: Plerixafor, Procedure: LeukapheresisParticipants in this study will receive a twice daily dose of Filgrastim (GCSF) (5 mcg/kg BID) SQ starting on day 1 for 5 days followed by a single dose of SQ plerixafor (0.24 mg/kg) on day 5 followed by collection of CD34+ HSPCs via apheresis. A portion of cells collected from the participant will be stored as backup to be used toward future gene therapy endeavors. The remaining cells will be donated for research studies |
Who may qualify
- Ages
- 18 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (11 listed)
- Participants with a bone marrow failure syndrome with an identified genetic cause willing to donate autologous HSPCs for advancing gene therapy
- Age ≥ 18 years - 25 years
- The following hematological parameters need to be met (regardless of transfusion or growth factor support)
- Bone marrow evaluation within the preceding 6 months prior to mobilization and apheresis
- Participants should either have a central venous catheter (CVC) in place, be able to undergo apheresis without requiring a CVC, or agree to having a temporary apheresis catheter placed
Key exclusion criteria (14 listed)
- Participant with sickle cell disease
- Participant who has had a prior autologous or allogeneic HSCT
- Active viral, bacterial, fungal, or parasitic infection
- Total bilirubin >2.5x ULN or transaminases >5x ULN
- Moderate or severe renal failure defined as serum/plasma creatinine >1.5 mg/dL and an estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 based on the CKD-Epi equation or the St. Jude equation
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 |
|---|---|---|
| FilgrastimAdministered twice daily dose starting on day 1 for 5 days. | Drug | BDSTEM Treatment |
| PlerixaforAdministered on day 5 via IV. | Drug | BDSTEM Treatment |
| LeukapheresisPeripheral venous access or through a central venous catheter approximately 4-5 hours after the dose of plerixafor is given. | Procedure or surgery | BDSTEM Treatment |
Primary outcome
Incidence of treatment-emergent adverse events following filgrastim plus plerixafor administration
Time frame: From initiation of drug administration through Day +7 to +10 follow-up
Safety will be assessed by the incidence, type, and severity of adverse events occurring after administration of filgrastim plus plerixafor in participants with bone marrow failure syndromes.
4 secondary outcomes
Number of participants achieving peripheral blood CD34+ counts ≥5 cells/µL
Time frame: From initiation of plerixafor administration through completion of apheresis, or 6 hours after drug administration if apheresis is not performed
Peripheral blood CD34+ kinetics following filgrastim plus plerixafor administration
Time frame: After plerixafor administration through completion of apheresis, or 6 hours after drug administration if apheresis is not performed
Observed CD34+ cell yield after 1 blood volume apheresis
Time frame: At completion of 1 blood volume apheresis on Day 5
Estimated total CD34+ cell yield from projected full-volume apheresis
Time frame: At completion of 1 blood volume apheresis on Day 5
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2026.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.
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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.
Alexis Leonard, MD principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Filgrastim, Plerixafor and Leukapheresis in people with Bone Marrow Failure Syndrome, run by St. Jude Children's Research Hospital 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 11 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 6 Oct 2026. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alexis Leonard, MD (888-226-4343, referralinfo@stjude.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events following filgrastim plus plerixafor administration (time frame: From initiation of drug administration through Day +7 to +10 follow-up). 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 6 Oct 2026.