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.
Brief summary
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.
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.
Trial details
- 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
Record
- Archive ID
- nct:NCT06672237
- Published
- Nov 4, 2024
- Updated
- Apr 16, 2026
- Venue
- ClinicalTrials.gov
- ClinicalTrials.gov
- NCT06672237
- Categories
- Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary, Neurodegenerative Diseases, Neuromuscular Diseases
- License
- ClinicalTrials.gov terms (attribution, processing date, modifications noted)
- Provenance
- clinicaltrials