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Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

NCT05329649 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 13

Last updated 2026-03-09

No results posted yet for this study

Summary

This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

Conditions

  • Sickle Cell Disease
  • Hydroxyurea Failure
  • Hydroxyurea Intolerance
  • Hemoglobinopathies
  • Hematological Diseases

Interventions

BIOLOGICAL

CTX001

Administered by intravenous infusion following myeloablative conditioning with busulfan.

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
11 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-05-02
Primary Completion
2026-05-31
Completion
2026-05-31
FDA Drug
Yes

Countries

  • United States
  • Germany
  • Italy
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05329649 on ClinicalTrials.gov