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NCT06506461 · ClinicalTrials.gov registry record · Phase 1
Gene Editing For Sickle Cell Disease
A Phase 1 study of Sickle Cell Disease, sponsored by St. Jude Children's Research Hospital.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 25
- Enrollment target
- 1
- Study location
NCT06506461: Recruiting Phase 1 study of Sickle Cell Disease, sponsored by St. Jude Children's Research Hospital.
NCT06506461 is a Phase 1 study of Sickle Cell Disease that is actively recruiting participants, run by St. Jude Children's Research Hospital. The registered enrollment target is 25 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (91% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06506461, a Phase 1 study of Sickle Cell Disease, is actively recruiting participants, sponsored by St. Jude Children's Research Hospital.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 25 participants
- Enrollment target
- 1
- Study location
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.
Primary Outcome
Upon completion of the trial, summary statistics will be computed for the time to neutrophil engraftment.
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 |
What NCT06506461 shows while recruiting
NCT06506461 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 25 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (91% lower).
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.
NCT06506461 reports a single indexed study location in Tennessee.
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.
What interventions are being tested in trial NCT06506461?
The interventions under investigation include: Busulfan (DRUG), Plerixafor (DRUG), Motixafortide (DRUG), Gene-modified CD34+ cells (BIOLOGICAL).
Who is sponsoring clinical trial NCT06506461?
This trial is sponsored by St. Jude Children's Research Hospital, which has 410 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
Similar trials for Sickle Cell Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06506461's enrollment target sits among peer trials
25 153rd of 213 higher than 55 of 213 other Sickle Cell Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Sickle Cell Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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