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Stem Cell Collection for Bone Marrow Failure Syndromes

This study aims to safely collect your own stem cells (HSPCs) if you have a bone marrow failure syndrome (a condition where your bone marrow doesn't make enough healthy blood cells). Researchers are testing two medications, Filgrastim and Plerixafor, to help move your stem cells from your bone marrow into your bloodstream. Once in your bloodstream, these cells will be collected through a procedure called leukapheresis. The main goal is to see how safe this process is for people with bone marrow failure syndromes. You can join if you are between 18 and 25 years old, have a bone marrow failure syndrome with a known genetic cause, and meet certain blood count requirements. The study plans to enroll 12 participants.

Study design
This is a Phase 1, open-label study, meaning both you and the study team will know which treatments you are receiving. It will involve 12 participants.
What's involved
You will have a screening period with tests, a physical exam, and a bone marrow evaluation. Then, you will receive medications and have a stem cell collection procedure, followed by outpatient follow-up and a phone call.
Compensation
Not stated in the trial record.
Follow-up
You will be followed up within 7-10 days after the stem cell collection, and again by phone about 30 days later. A bone marrow evaluation will also be done within 6 months after the procedure.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07585136

Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders

Not Yet Recruiting
PHASE1Ages 18–25Interventional
St. Jude Children's Research Hospital
~12 participants
Updated 2026-08-31 on ClinicalTrials.gov
What's tested:FilgrastimPlerixaforLeukapheresis

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of treatment-emergent adverse events following filgrastim plus plerixafor administration
Measured over From initiation of drug administration through Day +7 to +10 follow-up

Conditions

Bone Marrow Failure Syndrome

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Bone Marrow Failure Syndrome

NCT07585136

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Saint Jude Children's Research Hospital

    Memphis, Tennesseestudy coordinator listed

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Alexis Leonard, MD · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital

Who to contact

Alexis Leonard, MD
[email protected]
888-226-4343
Email the study team

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Eligibility criteria

Inclusion

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

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
Pregnancy
Inability or unwillingness of research participant to give written informed consent.

What this trial measures

  • Incidence of treatment-emergent adverse events following filgrastim plus plerixafor administrationFrom 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.