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Gene Editing For Sickle Cell Disease
NCT06506461 · View on ClinicalTrials.gov ↗
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 Studied
Interventions
- DRUG Busulfan
- DRUG Plerixafor
- DRUG Motixafortide
- BIOLOGICAL Gene-modified CD34+ cells
Study Locations (1)
Tennessee
- St. Jude Children's Research Hospital — Memphis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 25 participants |
| Start Date | 2025-03-21 |
| Est. Completion | 2032-12 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06506461
The ClinicalTrials.gov registry entry for NCT06506461 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 25 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is St. Jude Children's Research Hospital, which has 441 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Sickle Cell Disease appearing as the primary indexed condition, and to 4 interventions — of which Busulfan is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06506461 reports 1 study location spanning 1 distinct geographic area — top geographies include Tennessee. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06506461 about?
NCT06506461 is a clinical study titled "Gene Editing For Sickle Cell Disease". 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 sympto...
What is the current status of trial NCT06506461?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 25 participants. The study started on 2025-03-21. Estimated completion is 2032-12.
What conditions does trial NCT06506461 study?
This clinical trial studies the following conditions: Sickle Cell Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06506461?
The interventions under investigation include: Busulfan (DRUG), Plerixafor (DRUG), Motixafortide (DRUG), Gene-modified CD34+ cells (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06506461?
This trial is sponsored by St. Jude Children's Research Hospital, which has 441 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06506461 being conducted?
This trial has 1 study location across Tennessee. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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