Gene Editing For Sickle Cell Disease
Public ClinicalTrials.gov record NCT06506461. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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
- Enrollment
- 25 participants
Conditions and interventions
Conditions
Interventions
- Plerixafor Drug
- Busulfan Drug
- Gene-modified CD34+ cells Biological
- Motixafortide Drug
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 20, 2025
- Primary completion
- Nov 30, 2029
- Completion
- Nov 30, 2032
- Last update posted
- May 4, 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 May 4, 2026 · Synced Sep 23, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06506461 live on ClinicalTrials.gov.