A Phase 3 Study of NTLA-2001 in ATTRv-PN

ConditionsNeuromuscular Disease Neuromuscular Diseases (NMD)Neurodegenerative Disease Neurodegenerative Disease, Hereditary Neurodegenerative Diseases Neuromuscular Diseases Nerve Disorders Nervous System Disease Nervous System Diseases Genetic Disease, Inborn Amyloidosis, Familial Amyloidosis, Hereditary Amyloidosis Polyneuropathies Amyloid Neuropathies Amyloid Neuropathies, Familial Peripheral Nervous System Disease Peripheral Nervous System Diseases Metabolism, Inborn Errors Metabolic Diseases

Trial statusRecruiting

Trial phasePhase 3

Trial typeInterventional

Biological sexAll

Age18-85

SponsorIntellia Therapeutics

About this trial

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.

Eligibility criteria

Qualifiers

Diagnosis of ATTRv-PN

Karnofsky Performance Status (KPS) ≥ 60

Disqualifiers

Other causes of amyloidosis (amyloidosis caused by non-TTR protein)

Other known causes of sensorimotor or autonomic neuropathy

Diabetes mellitus

New York Heart Association Class III or IV heart failure

Trial design

Treatments tested in this trial

nexiguran ziclumeran
Normal Saline as Placebo

Treatment groups

60 Participants

are divided into 2 treatment groups

Locations

Argentina

Hospital Británico de Buenos Aires Buenos Aires Argentina

Hospital Italiano de Buenos Aires (HIBA) Buenos Aires Argentina

Hospital El Cruce San Juan Bautista Argentina

Brazil

Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP) Campinas Brazil

Sponsors and collaborators

Intellia Therapeutics

Lead sponsor

Regeneron Pharmaceuticals

Collaborator

Source ClinicalTrials.gov