Sickle Cell Disease and β-Thalassemia: CTX001 Treatment Study

We are studying the long-term safety and effectiveness of a new CRISPR-based treatment for patients with β-thalassemia or severe sickle cell disease. This research aims to understand how well the treatment works over time.

Confirmation phase (III)No Placebo Cost Reimbursement Partially Remote Standard Medicines InjectionHematology

What diseases are being studied?

Health conditions and diseases that the clinical trial is designed to study and treat.

What medicines are being studied?

Medicines that are officially approved for use in the European Union and considered safe by EMA. These medicines are already on the market and may be tested for new conditions or populations.

Casgevy

Casgevy is a one-time gene-editing cell therapy used to treat severe sickle cell disease and transfusion-dependent beta-thalassemia.

What active substances are being studied?

Active substances that are officially approved for use in the European Union and considered safe by EMA. These substances are already on the market and may be tested for new conditions or populations.

Exagamglogene Autotemcel

Exagamglogene autotemcel is a gene-edited stem cell therapy that helps people with certain inherited blood disorders produce healthier red blood cells.

Where is the clinical trial being conducted?

Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.

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Association Hospitaliere De Bruxelles Hopital Universitaire Des Enfants Reine Fabiola

Pediatric Hemato-Oncology

Jette, Belgium

Universitaetsklinikum Duesseldorf AöR

Department of Pediatric Oncology, Hematology and Clinical Immunology

Düsseldorf, Germany

Universitaetsklinikum Regensburg AöR

Pediatric Hematology, Oncology and Stem cell transplantation

Pentling, Germany

Sponsor: Vertex Pharmaceuticals Inc.

Last updated: Nov 27, 2025

Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.