A Long-term Follow-up Study in Participants Who Received CTX001
Enrolling by invitation · Phase 3
Conditions studied: Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
In brief
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).
Key facts
- Study ID
- NCT04208529
- Run by
- Vertex Pharmaceuticals Incorporated
- People needed
- 160
- Starts
- 2021-01-20
- Expected to finish
- 2039-09-30
- Last updated by the study team
- 2026-03-25
Who can join
Age: 2 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form
- Participants must have received CTX001 infusion in a parent study
You may not qualify if…
- There are no exclusion criteria
Where it is running
- 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
Full record on ClinicalTrials.gov
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