Sickle Cell Disease: CTX001 Treatment for Kids

We are studying a new gene-editing treatment for children with severe sickle cell disease. The trial aims to evaluate its safety and effectiveness in improving their health.

>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.
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.
Plerixafor
Plerixafor is a substance that helps move blood-forming stem cells into the bloodstream for collection before stem cell transplant.

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.

Mozobil
Pleforbil
Pleriksafor Onkogen

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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Universitaetsklinikum Duesseldorf AöR
Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
Ospedale Pediatrico Bambino Gesu
Department of Pediatric Hematology and Oncology IRCCS
Rome, Italy
Sponsor: Vertex Pharmaceuticals Inc.
Last updated: Nov 26, 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.