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Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease (SCD) Using Plerixafor

NCT04817345 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2023-04-07

No results posted yet for this study

Summary

With recent advances in gene editing, gene therapy is becoming a viable curative treatment option for sickle cell disease. In order to do genetic manipulation, investigators need to collect hematopoietic stem cells from patients with sickle cell disease. In this study, investigators want to study the safety and feasibility of collecting peripheral blood stem cells from pediatric and young adult patients with sickle cell disease after administering plerixafor. Studying these peripheral blood stem cells will help in optimizing the yield of peripheral CD34+ cells from pediatric and young adult patients with sickle cell disease, which in turn will help to develop better gene therapies for these patients.

Primary Objectives

* Determine the safety profile associated with administration of plerixafor in pediatric and young adult patients with sickle cell disease (SCD).
* To estimate the number of CD34+ cells/kg of body weight that can be collected with peripheral apheresis after administration of plerixafor in pediatric and young adult patients with SCD.

Exploratory Objectives

* To describe the kinetics of CD34+ cell mobilization in peripheral blood after - + cells obtained from pediatric and young adult patients with SCD.
* To study the effect of hydroxyurea therapy on senescence in plerixafor-mobilized CD34+ cells obtained from pediatric and young adult patients with SCD.

Conditions

Interventions

DRUG

Plerixafor

Subcutaneous

Sponsors & Collaborators

Principal Investigators

  • Akshay Sharma, MBBS · St. Jude Children's Research Hospital

  • Mitch Weiss, MD, PhD · St. Jude Children's Research Hospital

Study Design

Allocation
NA
Purpose
SUPPORTIVE_CARE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2023-04-30
Primary Completion
2023-04-30
Completion
2023-04-30
FDA Drug
Yes

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Diseases
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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 NCT04817345 on ClinicalTrials.gov