Clinical Trial

Disease: Sickle Cell Disease, SCD, (NCT05329649)

Disease info:

Sickle cell disease is a group of disorders that affects haemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical haemoglobin molecules called haemoglobin S, which can distort red blood cells into a sickle or crescent shape.

The production of haemoglobin A, which is the principle type of haemoglobin in humans, is governed by 3 genes: HBA1, HBA2, and HBB. Each haemoglobin A molecule consists of two alpha and two beta chains, and mutations in either of the HBA or the HBB genes may result in abnormal haemoglobin molecules with reduced or diminshed function. Sickle cell diseaase arises from a single point mutation in the 6th codon of the beta-globin gene (HBB), which results in a valine instead of a glutamic acid in the haemoglobin beta-chain.

Abnormal haemoglobin ultimately leads to anaemia as well as other symptoms, depending on the exact mutations present. Diseases caused by defective haemoglobin fall into a larger category of diseases known as the "haemoglobinopathies" which also include the thalassemias, a related group of diseases that are characterised by reduced or deficient rather than abnormal haemoglobin. 

Sickle cell disease affects approximately 100,000 individuals in the USA and more than 3 million worldwide
Official title:
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease

Contact Information

Phone: 617-341-6777



United States, North Carolina

Atrium Health Levine Children's Hospital, Charlotte, North Carolina, United States, 28203


United States, Pennsylvania

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104


United States, Tennessee

St. Jude Children's Research Hospital, Memphis, Tennessee, United States, 38105

The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers, Nashville, Tennessee, United States, 37203

Germany, Dusseldorf
University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology, Dusseldorf, Germany


Italy, Rome

Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS, Rome, Italy


United Kingdom

St Mary's Hospital, London, United Kingdom

Study start:
May. 2, 2022
Gene editing method:
Type of edit:
Gene disruption
BAF Chromatin Remodeling Complex Subunit 11A (BCL11A)
Delivery method:
Electroporation - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


This is a single-dose, open-label study in paediatric 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 haematopoietic stem and progenitor cells (hHSPCs) (CTX001).

Last updated: Mar. 22, 2024
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