Recruiting
Phase 1

Filgrastim & Plerixafor

Sponsor:

St. Jude Children's Research Hospital

Code:

NCT07585136

Conditions

Bone Marrow Failure Syndrome

Eligibility Criteria

Sex: All

Age: 18 - 25

Healthy Volunteers: Not accepted

Interventions

Filgrastim

Plerixafor

Leukapheresis

Study Details

Brief 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.

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).

Conditions

Bone Marrow Failure Syndrome

Study ID

NCT07585136

Start date

Nov, 2026

Status verified date

Oct, 2026

Completion date

Jul 1, 2029

Anticipated

Primary completion date

Dec 1, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 25

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

12 participants

Anticipated

Intervention Model

Single group

Primary purpose

Other

Interventions and Outcome Measures

Arms

experimental: BDSTEM Treatment

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

Interventions

Filgrastim

Administered twice daily dose starting on day 1 for 5 days.

Plerixafor

Administered on day 5 via IV.

Leukapheresis

Peripheral venous access or through a central venous catheter approximately 4-5 hours after the dose of plerixafor is given.

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 ]

Central Contacts and Locations

Central contacts

Alexis Leonard, MD

888-226-4343referralinfo@stjude.org

Locations

Saint Jude Children's Research Hospital

Recruiting

Memphis, Tennessee, United States, 38105-2794

Contacts

Alexis Leonard, MD

888-226-4343referralinfo@stjude.org

More Information

Sponsor

St. Jude Children's Research Hospital

Last update posted

Oct 6, 2026

Last verified

Oct, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-07. This information was provided to ClinicalTrials.gov by St. Jude Children's Research Hospital on 2026-10-06. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.