{"id":"nct:NCT05951205","kind":"trial","title":"Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype","authors":["Vertex Pharmaceuticals Incorporated"],"author_count":1,"year":2023,"published":"2023-07-18","venue":"ClinicalTrials.gov","cited_by":null,"doi":null,"arxiv_id":null,"nct_id":"NCT05951205","source_url":"https://clinicaltrials.gov/study/NCT05951205","archive_url":"https://space-kitty.com/papers/nct:NCT05951205","updated":"2026-03-25","pmid":null,"pmcid":null,"openalex_id":null,"pdf_url":null,"abstract":"The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).","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":["Sickle Cell Disease"],"details":{"acronym":null,"status":"WITHDRAWN","phase":"PHASE3","study_type":"INTERVENTIONAL","enrollment":0,"sponsor":"Vertex Pharmaceuticals Incorporated","conditions":["Sickle Cell Disease"],"interventions":["BIOLOGICAL: Exa-cel"],"start_date":"2027-07-31","start_date_type":"ESTIMATED","primary_completion_date":"2033-12-31","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":[]}