{"id":"nct:NCT06672237","kind":"trial","title":"A Phase 3 Study of NTLA-2001 in ATTRv-PN","authors":["Intellia Therapeutics"],"author_count":1,"year":2024,"published":"2024-11-04","venue":"ClinicalTrials.gov","cited_by":null,"doi":null,"arxiv_id":null,"nct_id":"NCT06672237","source_url":"https://clinicaltrials.gov/study/NCT06672237","archive_url":"https://space-kitty.com/papers/nct:NCT06672237","updated":"2026-04-16","pmid":null,"pmcid":null,"openalex_id":null,"pdf_url":null,"abstract":"This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.","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":["Neuromuscular Disease","Neuromuscular Diseases (NMD)","Neurodegenerative Disease","Neurodegenerative Disease, Hereditary","Neurodegenerative Diseases","Neuromuscular Diseases"],"details":{"acronym":null,"status":"RECRUITING","phase":"PHASE3","study_type":"INTERVENTIONAL","enrollment":60,"sponsor":"Intellia Therapeutics","conditions":["Neuromuscular Disease","Neuromuscular Diseases (NMD)","Neurodegenerative Disease","Neurodegenerative Disease, Hereditary","Neurodegenerative Diseases","Neuromuscular Diseases","Nerve Disorders","Nervous System Disease","Nervous System Diseases","Genetic Disease, Inborn"],"interventions":["BIOLOGICAL: nexiguran ziclumeran","DRUG: Normal Saline as Placebo"],"start_date":"2024-11-22","start_date_type":"ACTUAL","primary_completion_date":"2027-07-01","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"}]}