A Long-term Follow-up Study in Participants Who Received CTX001
NCT ID: NCT04208529
Last Updated: 2025-11-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
PHASE3
160 participants
INTERVENTIONAL
2021-01-20
2039-09-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
OTHER
NONE
Study Groups
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CTX001
All participants who complete or discontinue one of the multiple parent studies (CTX001-111, CTX001-121, CTX001-141, CTX001-151, CTX001-161 and CTX001-171) after CTX001 infusion will be asked to participate in this long-term follow-up study.
CTX001
CTX001 infusion.
Interventions
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CTX001
CTX001 infusion.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Participants must have received CTX001 infusion in a parent study
2 Years
ALL
No
Sponsors
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CRISPR Therapeutics
INDUSTRY
Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Locations
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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
Hammersmith Hospital - Haematology Dept
London, , United Kingdom
University College London Hospital NHS Foundation - Main
London, , United Kingdom
Countries
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References
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de la Fuente J, Frangoul H, Lang P, Wall D, Meisel R, Corbacioglu S, Li AM, Shah AJ, Carpenter B, Kwiatkowski JL, Mapara MY, Liem RI, Rupprecht J, Kuo KHM, Merkeley H, Algeri M, Smith W, Kohli P, Li N, Rubin J, Zhang S, Hobbs WE, Locatelli F. Improvements in Health-Related Quality of Life in Patients with Transfusion-Dependent beta-Thalassemia After Exagamglogene Autotemcel. Blood Adv. 2025 Aug 19:bloodadvances.2025016702. doi: 10.1182/bloodadvances.2025016702. Online ahead of print.
Sharma A, Locatelli F, Bhatia M, Molinari L, Mapara MY, Liem RI, Dedeken L, Wall D, Eckrich MJ, Kuo KHM, Smith W, Imren S, Kohli P, Li N, Liu T, Rubin J, Hobbs WE, Grupp SA, Frangoul H. Improvements in Health-Related Quality of Life in Patients with Severe Sickle Cell Disease After Exagamglogene Autotemcel. Blood Adv. 2025 Aug 19:bloodadvances.2025016701. doi: 10.1182/bloodadvances.2025016701. Online ahead of print.
Other Identifiers
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2024-512654-19-00
Identifier Type: OTHER
Identifier Source: secondary_id
VX18-CTX001-131
Identifier Type: -
Identifier Source: org_study_id
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