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NCT03745287

A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

Study Summary

This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with 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.

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Interventions

CTX001BIOLOGICAL
Administered by IV infusion following myeloablative conditioning with busulfan.

Study Locations

FacilityCityStateCountry
Lucile Packard Children's Hospital of Stanford UniversityPalo AltoCaliforniaUnited States
Ann & Robert Lurie Children's Hospital of ChicagoChicagoIllinoisUnited States
University of Illinois at Chicago Hospitals and Health SystemsChicagoIllinoisUnited States
Columbia University Medical Center (21+ years)New YorkNew YorkUnited States
Columbia University Medical Center (≤21 years)New YorkNew YorkUnited States
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvaniaUnited States
St. Jude Children's Research HospitalMemphisTennesseeUnited States
The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood CancersNashvilleTennesseeUnited States
Methodist Children's Hospital/Texas Transplant InstituteSan AntonioTexasUnited States
Hopital Universitaire des Enfants Reine Fabiola (HUDERF)BrusselsBelgium
The Hospital for Sick ChildrenTorontoCanada
Hopital Necker Enfants MaladesParisFrance
University Hospital DuesseldorfDüsseldorfGermany
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Paediatric Haemotology, Oncology and Stem Cell TransplantationRegensburgGermany
Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCSRomeItaly
Imperial College Healthcare NHS Trust, Hammersmith HospitalLondonUnited Kingdom
Royal London and St Bartholomew's Hospital, Pathology and Pharmacy BuildingLondonUnited Kingdom

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026