Gene Editing For Sickle Cell Disease

ClinicalTrials.gov ID: NCT06506461

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ClinicalTrials.gov public records Last synced Nov 14, 2026, 9:47 PM EST

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Official title

St. Jude Autologous Genome Edited Stem Cells For Sickle Cell Disease-1

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID: NCT06506461
Recruitment status: Recruiting
Study type: Interventional
Phase: Phase 1
Lead sponsor: St. Jude Children's Research Hospital; Other
Enrollment: 25 participants

Conditions and interventions

Conditions

Sickle Cell Disease

Interventions

Drug · Biological

Eligibility (public fields only)

Age range: 18 Years to 24 Years
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Mar 21, 2025 (Actual)
Primary completion: Dec 2029 (Estimated)
Completion: Dec 2032 (Estimated)
Last update posted: Oct 15, 2026

2025 – 2032

United States locations

U.S. sites: 1
U.S. states: 1
U.S. cities: 1

Facility	City	State	ZIP	Site status
St. Jude Children's Research Hospital	Memphis	Tennessee	38105	Recruiting

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT06506461, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Oct 15, 2026 · Synced Nov 14, 2026