Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders

NCT07585136 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: BDSTEM
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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 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).

Conditions

Bone Marrow Failure Syndrome

Outcome Measures

Primary Outcomes (1)

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

  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.

  From initiation of drug administration through Day +7 to +10 follow-up

Secondary Outcomes (4)

• Number of participants achieving peripheral blood CD34+ counts ≥5 cells/µL

  From initiation of plerixafor administration through completion of apheresis, or 6 hours after drug administration if apheresis is not performed

• Peripheral blood CD34+ kinetics following filgrastim plus plerixafor administration

  After plerixafor administration through completion of apheresis, or 6 hours after drug administration if apheresis is not performed

• Observed CD34+ cell yield after 1 blood volume apheresis

  At completion of 1 blood volume apheresis on Day 5

• Estimated total CD34+ cell yield from projected full-volume apheresis

  At completion of 1 blood volume apheresis on Day 5

Study Arms (1)

BDSTEM Treatment

EXPERIMENTAL

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

Drug: Filgrastim; Drug: Plerixafor; Procedure: Leukapheresis

Interventions

Filgrastim DRUG

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

BDSTEM Treatment

Plerixafor DRUG

Administered on day 5 via IV.

BDSTEM Treatment

Leukapheresis PROCEDURE

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

BDSTEM Treatment

Eligibility Criteria

• Age: 18 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

Saint Jude Children's Research Hospital

Memphis, Tennessee, 38105-2794, United States

Location

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

MeSH Terms

Conditions

Bone Marrow Failure Disorders

Interventions

Filgrastim; plerixafor; Leukapheresis

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Cytapheresis; Biological Therapy; Therapeutics; Blood Component Removal; Leukocyte Reduction Procedures; Cell Separation; Cytological Techniques; Clinical Laboratory Techniques; Investigative Techniques

Study Officials

• Alexis Leonard, MD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Alexis Leonard, MD

CONTACT

888-226-4343; referralinfo@stjude.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 16, 2026
• First Posted: May 13, 2026
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): July 1, 2029
• Last Updated: May 22, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data will be made available at the time of article publication.
• Access Criteria: Data will be provided to researchers following a formal request with the following information: full name of requestor, affiliation, data set requested, and timing of when data is needed. As an informational point, the lead statistician and study principal investigator will be informed that primary results datasets have been requested.

Locations

US (1)