{"id":"nct:NCT04208529","kind":"trial","title":"A Long-term Follow-up Study in Participants Who Received CTX001","authors":["Vertex Pharmaceuticals Incorporated"],"author_count":1,"year":2019,"published":"2019-12-23","venue":"ClinicalTrials.gov","cited_by":null,"doi":null,"arxiv_id":null,"nct_id":"NCT04208529","source_url":"https://clinicaltrials.gov/study/NCT04208529","archive_url":"https://space-kitty.com/papers/nct:NCT04208529","updated":"2026-08-11","pmid":null,"pmcid":null,"openalex_id":null,"pdf_url":null,"abstract":"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).","abstract_license":"ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters","license":"ClinicalTrials.gov terms (attribution, processing date, modifications noted)","is_open_access":true,"retracted":false,"peer_reviewed":null,"categories":["Beta-Thalassemia","Thalassemia","Sickle Cell Disease","Hematologic Diseases","Hemoglobinopathies","Genetic Diseases, Inborn"],"details":{"acronym":null,"status":"ENROLLING_BY_INVITATION","phase":"PHASE3","study_type":"INTERVENTIONAL","enrollment":160,"sponsor":"Vertex Pharmaceuticals Incorporated","conditions":["Beta-Thalassemia","Thalassemia","Sickle Cell Disease","Hematologic Diseases","Hemoglobinopathies","Genetic Diseases, Inborn","Sickle Cell Anemia"],"interventions":["BIOLOGICAL: CTX001"],"start_date":"2021-01-20","start_date_type":"ACTUAL","primary_completion_date":"2039-09-30","has_results":false,"data_source":"ClinicalTrials.gov","data_processed":"2026-10-29T09:00:05","modifications":"selected fields only; whitespace normalized; brief summary truncated to 2,000 characters"},"provenance":"clinicaltrials","attribution":"Source: ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters. Not a U.S. Government endorsement.","topics":[{"slug":"gene-editing-therapies","name":"CRISPR & Gene-Editing Therapies","domain_slug":"medicine"}],"cited_in_dossiers":[{"slug":"gene-editing-therapies","title":"CRISPR & Gene-Editing Therapies: State of the Field (October 2026)","url":"https://space-kitty.com/dossiers/gene-editing-therapies"}]}