A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
NCT ID: NCT03655678
Last Updated: 2025-12-17
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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COMPLETED
PHASE2/PHASE3
59 participants
INTERVENTIONAL
2018-09-14
2025-11-13
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
TREATMENT
NONE
Study Groups
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CTX001
CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive a single infusion of CTX001 through a central venous catheter.
CTX001
Administered by IV infusion following myeloablative conditioning with busulfan
Interventions
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CTX001
Administered by IV infusion following myeloablative conditioning with busulfan
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
1. Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning
2. History of at least 100 mL/kg/year or ≥10 units/year of packed RBC transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening
* Eligible for autologous stem cell transplant as per investigator's judgment
Exclusion Criteria
* Prior allo-HSCT
* Participants with associated α-thalassemia and \>1 alpha deletion or alpha multiplications
* Participants with sickle cell beta thalassemia variant
* Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator
* White blood cell (WBC) count \<3 × 10\^9/L or platelet count \<50 × 10\^9/L not related to hypersplenism
12 Years
35 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 Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Columbia University Medical Center (21+ years)
New York, New York, United States
Columbia University Medical Center
New York, New York, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers
Nashville, Tennessee, United States
The Hospital for Sick Children
Toronto, , Canada
British Columbia Children's Hospital
Vancouver, , Canada
Universitätsklinikum Düsseldorf Hospital Duesseldorf
Düsseldorf, , Germany
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine
Regensburg, , Germany
University Hospital Tübingen
Tübingen, , Germany
Ospedale Pediatrico Bambino Gesù, IRCCS
Rome, , Italy
Imperial College Healthcare NHS Trust, Hammersmith Hospital
London, , United Kingdom
University College London Hospitals NHS Foundation Trust
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.
Locatelli F, Lang P, Wall D, Meisel R, Corbacioglu S, Li AM, de la Fuente J, Shah AJ, Carpenter B, Kwiatkowski JL, Mapara M, Liem RI, Cappellini MD, Algeri M, Kattamis A, Sheth S, Grupp S, Handgretinger R, Kohli P, Shi D, Ross L, Bobruff Y, Simard C, Zhang L, Morrow PK, Hobbs WE, Frangoul H; CLIMB THAL-111 Study Group. Exagamglogene Autotemcel for Transfusion-Dependent beta-Thalassemia. N Engl J Med. 2024 May 9;390(18):1663-1676. doi: 10.1056/NEJMoa2309673. Epub 2024 Apr 24.
Brusson M, Miccio A. Genome editing approaches to beta-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.
Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and beta-Thalassemia. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5.
Other Identifiers
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CTX001-111
Identifier Type: -
Identifier Source: org_study_id
2024-516894-57-00
Identifier Type: OTHER
Identifier Source: secondary_id