A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

Study Summary

This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649) or 161(NCT05477563) (severe sickle cell disease \[SCD\] studies).

Interventions

Study Locations

Facility	City	State	Country
Lucile Packard Children's Hospital	Palo Alto	California	United States
Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology	Chicago	Illinois	United States
Herbert Irving Pavilion - Hematology	New York	New York	United States
New York Presbyterian Hospital - Morgan Stanley Children's Hospital	New York	New York	United States
Levine Children's Hospital - Hematology	Charlotte	North Carolina	United States
The Children's Hospital of Philadelphia - Hematology	Philadelphia	Pennsylvania	United States
St. Jude Children's Research Hospital	Memphis	Tennessee	United States
TriStar Medical Group Children's Specialists - Pediatric Oncology	Nashville	Tennessee	United States
Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital	San Antonio	Texas	United States
Hopital Universitaire des Enfants Reine Fabiola (HUDERF) - Hematology	Brussels		Belgium
Hospital for Sick Children - Hematology	Toronto		Canada
Toronto General Hospital - Hematology	Toronto		Canada
St. Paul's Hospital - Hematology	Vancouver		Canada
University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology	Düsseldorf		Germany
Center for Pediatric Clinical Studies (CPCS)	Klinik Für Kinder- Und Jugendmedizin		Germany
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine	Regensburg		Germany
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica	Rome		Italy
Great Ormond Street Hospital for Children	London		United Kingdom
Hammersmith Hospital - Haematology Dept	London		United Kingdom
University College London Hospital NHS Foundation - Main	London		United Kingdom

Official Trial Information

View on ClinicalTrials.gov