Transfusion-Dependent β-Thalassemia: CTX001 Treatment

We are studying a new cell therapy for children with transfusion-dependent β-thalassemia to see if it can improve their condition and reduce the need for blood transfusions. The trial will also assess the safety of this treatment.

>2 yearsConfirmation phase (III)Post-Trial Drug AccessNo PlaceboCost ReimbursementStandard MedicinesInjectionHematologyPediatrics

What diseases are being studied?

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

Who can participate in the clinical trial?

These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..

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.
Filgrastim Hexal
Filgrastim Hexal is a medicine that boosts white blood cell production to help reduce infection risk during chemotherapy or after bone marrow transplant.
Plerixafor
Plerixafor helps move blood-forming stem cells into the bloodstream so they can be collected for transplant in certain blood cancers.

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.

Busulfan
Busulfan is a substance used to destroy bone marrow cells before a bone marrow transplant and to treat certain blood cancers.
Exagamglogene Autotemcel
Exagamglogene autotemcel is a gene-edited stem cell therapy that helps people with certain inherited blood disorders produce healthier red blood cells.
Filgrastim
Filgrastim is a substance that stimulates production of neutrophils, a type of white blood cell, to help prevent infections in people with low counts.

What similar medicines might you know?

Don't worry if you don't know the medicines tested in the trial. There is a chance you have heard about the similar medicines. Check the list below to see if you are familiar with any of them.

Accofil
Granulokine
Grastofil

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.

Loading map...
Universitaetsklinikum Duesseldorf AöR
Clinic for pediatric oncology, hematology and clinical immunology
Düsseldorf, Germany
Bambino Gesu Childrens Hospital
Department of Pediatric Hematology and Oncology IRCCS
Rome, Italy
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.