{"id":"nct:NCT03655678","kind":"trial","title":"A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia","authors":["Vertex Pharmaceuticals Incorporated"],"author_count":1,"year":2018,"published":"2018-08-31","venue":"ClinicalTrials.gov","cited_by":null,"doi":null,"arxiv_id":null,"nct_id":"NCT03655678","source_url":"https://clinicaltrials.gov/study/NCT03655678","archive_url":"https://space-kitty.com/papers/nct:NCT03655678","updated":"2025-12-17","pmid":null,"pmcid":null,"openalex_id":null,"pdf_url":null,"abstract":"This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.","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","Genetic Diseases, Inborn","Hematologic Diseases","Hemoglobinopathies"],"details":{"acronym":null,"status":"COMPLETED","phase":"PHASE2, PHASE3","study_type":"INTERVENTIONAL","enrollment":59,"sponsor":"Vertex Pharmaceuticals Incorporated","conditions":["Beta-Thalassemia","Thalassemia","Genetic Diseases, Inborn","Hematologic Diseases","Hemoglobinopathies"],"interventions":["BIOLOGICAL: CTX001"],"start_date":"2018-09-14","start_date_type":"ACTUAL","primary_completion_date":"2025-11-13","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":[]}