{"id":"nct:NCT05477563","kind":"trial","title":"Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease","authors":["Vertex Pharmaceuticals Incorporated"],"author_count":1,"year":2022,"published":"2022-07-28","venue":"ClinicalTrials.gov","cited_by":null,"doi":null,"arxiv_id":null,"nct_id":"NCT05477563","source_url":"https://clinicaltrials.gov/study/NCT05477563","archive_url":"https://space-kitty.com/papers/nct:NCT05477563","updated":"2026-03-23","pmid":null,"pmcid":null,"openalex_id":null,"pdf_url":null,"abstract":"This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). 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","Hematologic Diseases","Genetic Diseases, Inborn","Hemoglobinopathies","Sickle Cell Disease"],"details":{"acronym":null,"status":"RECRUITING","phase":"PHASE3","study_type":"INTERVENTIONAL","enrollment":26,"sponsor":"Vertex Pharmaceuticals Incorporated","conditions":["Beta-Thalassemia","Thalassemia","Hematologic Diseases","Genetic Diseases, Inborn","Hemoglobinopathies","Sickle Cell Disease","Sickle Cell Anemia"],"interventions":["BIOLOGICAL: CTX001"],"start_date":"2022-08-02","start_date_type":"ACTUAL","primary_completion_date":"2027-06-09","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":[]}