NCT07585136 · St. Jude Children's Research Hospital
Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders
What this study is about
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.
View original scientific description
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).
Interventions
DRUG
Filgrastim
Administered twice daily dose starting on day 1 for 5 days.
DRUG
Plerixafor
Administered on day 5 via IV.
PROCEDURE
Leukapheresis
Peripheral venous access or through a central venous catheter approximately 4-5 hours after the dose of plerixafor is given.
Primary outcome measures
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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
- 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
Exclusion criteria
- 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
- 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
- Inability or unwillingness of research participant to give written informed consent.
Where
- Memphis, Tennessee
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Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Oct 6, 2026 · Source of record for eligibility and locations