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Gene Editing For Sickle Cell Disease

NCT06506461 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2026-05-05

No results posted yet for this study

Summary

This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD.

Primary Objective

* To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.

Secondary Objective

* To assess the efficacy autologous infusion of CRISPR/Cas9 genome-edited CD34+ HSPCs into patients with severe SCD.

Conditions

Interventions

DRUG

Plerixafor

Given Subcutaneous (under the skin)

DRUG

Busulfan

Given Intravenous (IV)

BIOLOGICAL

Gene-modified CD34+ cells

Given Intravenous (IV)

DRUG

Motixafortide

Given Subcutaneous (under the skin)

Sponsors & Collaborators

Principal Investigators

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

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2025-03-21
Primary Completion
2029-12-31
Completion
2032-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases
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 NCT06506461 on ClinicalTrials.gov