Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

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 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.

View on ClinicalTrials.gov ↗

View your shortlist →

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.

Conditions Studied

Sickle Cell Disease

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

FieldValue
Enrollment Target 25 participants
Start Date 2025-03-21
Est. Completion 2032-12
Phase Phase 1

Sponsor

St. Jude Children's Research Hospital

410 total trials

Interested in This Trial?

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, categorized as Phase 1. The registered enrollment target is 25 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (91% lower). The listed sponsor is St. Jude Children's Research Hospital, which has 410 total studies on file at ClinicalTrials.gov.

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 1 study location spanning 1 distinct geographic area - top geographies include 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.

Explore more on PlainTrial

Sickle Cell Disease

All registered trials for this condition

Tennessee

All trials with a study site in Tennessee

Recruiting now

Find trials open to new participants near you

Rankings

Top sponsors, conditions, and phases

Read our methodology - how this data is sourced, computed, and verified.

Related

Browse all → | Rankings →
Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.