Stem cell mobilization and collection for life-threatening blood disorders

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a phase 1, open-label, single-center study to evaluate whether Filgrastim and plerixafor can safely mobilize hematopoietic stem/progenitor cells (HSPCs) and allow their collection via leukapheresis in patients with bone marrow failure syndromes. The trial includes screening, mobilization and collection, and short- and medium-term follow-up, with safety as the primary focus and measures of stem cell yield as secondary endpoints. It assesses adverse events and the peak CD34+ cell counts in peripheral blood, along with the feasibility and yield of CD34+ cells (CD34+ cells per kg). Enrollment targets adults with confirmed genetic BMFS who meet hematologic criteria and can undergo apheresis, with post-procedure marrow evaluation planned within 6 months.

Why it matters

Who can join: the full rules

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

Where it's running

• Saint Jude Children's Research Hospital
  Memphis, Tennessee, United States Recruiting

Study contacts

• Principal investigator: Alexis Leonard, MD — St. Jude Children's Research Hospital
• Study coordinator: Alexis Leonard, MD
• Email: referralinfo@stjude.org
• Phone: 888-226-4343

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.