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Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders

NCT07585136 · Status: NOT_YET_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-05-22

No results posted yet for this study

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

Interventions

DRUG

Filgrastim

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

DRUG

Plerixafor

Administered on day 5 via IV.

PROCEDURE

Leukapheresis

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

Sponsors & Collaborators

Principal Investigators

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

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-30
Primary Completion
2028-12-01
Completion
2029-07-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07585136 on ClinicalTrials.gov