Gene Editing For Sickle Cell Disease

Part of paid clinical trials in Memphis, Tennessee.

Sponsor
St. Jude Children's Research Hospital
Study ID
NCT06506461
Phase
PHASE1
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 24 Years
Healthy Volunteers
Not accepted

Interventions

  • Plerixafor — DRUG
    Given Subcutaneous (under the skin)
  • Busulfan — DRUG
    Given Intravenous (IV)
  • Gene-modified CD34+ cells — BIOLOGICAL
    Given Intravenous (IV)
  • Motixafortide — DRUG
    Given Subcutaneous (under the skin)

Study Details

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.

Key Dates

Start date
Mar 21, 2025
Status verified
Apr 2026
Primary completion
Dec 31, 2029
Completion
Dec 31, 2032

Study Design

Enrollment
25 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Autologous, genetically modified CD34+ HSPCs Treatment
    All eligible participants receive intervention as described in the Detailed Description with the following: motixafortide, plerixafor, busulfan, and autologous, gene-modified CD34+ cells

Primary Outcome Measure

Incidence of neutrophil engraftment by day +42 after infusion of the CRISPR/Cas9-edited CD34+ HSPCs. [ Time Frame: Within 42 days of the cellular product infusion ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
St. Jude Children's Research HospitalMemphisTennessee38105
Akshay Sharma, MBBS, MSc
[email protected]
888-226-4343

Find similar trials in Memphis, TN

By condition
Sickle cell disease
By research site
St. Jude Children's Research Hospital· Memphis, TN

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