Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders

Part of paid clinical trials in Memphis, Tennessee.

Sponsor
St. Jude Children's Research Hospital
Study ID
NCT07585136
Phase
PHASE1
Status
Not Yet Recruiting

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Conditions

  • Bone Marrow Failure Syndrome

Eligibility Criteria

Sex
ALL
Age
18 Years - 25 Years
Healthy Volunteers
Not accepted

Interventions

  • Filgrastim — DRUG
    Administered twice daily dose starting on day 1 for 5 days.
  • Plerixafor — DRUG
    Administered on day 5 via IV.
  • Leukapheresis — PROCEDURE
    Peripheral venous access or through a central venous catheter approximately 4-5 hours after the dose of plerixafor is given.

Study Details

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 bone marrow failure syndromes as determined by the incidence of adverse events (AEs). Secondary Objectives: * To characterize the feasibility of HSPC mobilization using Filgrastim plus plerixafor as determined by peripheral blood CD34+ counts. * To measure the mobilization effects of Filgrastim plus plerixafor in the peripheral blood in participants as determined by peak 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).

Key Dates

Start date
Jun 30, 2026
Status verified
May 2026
Primary completion
Dec 1, 2028
Completion
Jul 1, 2029

Study Design

Enrollment
12 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER

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

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

Locations (1)

FacilityCityStateZIPSite coordinators
Saint Jude Children's Research HospitalMemphisTennessee38105-2794
Alexis Leonard, MD
[email protected]
888-226-4343

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By research site
Saint Jude Children's Research Hospital· Memphis, TN

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