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Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT05477563 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 26

Last updated 2026-03-23

No results posted yet for this study

Summary

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.

Conditions

Interventions

BIOLOGICAL

CTX001

Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-02
Primary Completion
2027-06-09
Completion
2027-06-09
FDA Drug
Yes

Countries

  • United States
  • Germany
  • Italy
  • Saudi Arabia

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 NCT05477563 on ClinicalTrials.gov