NCT06672237

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

MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Intellia Therapeutics · Industry
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 60 participants, who will be randomized 1:1 to receive a single infusion of either nexiguran ziclumeran or placebo. To ensure all participants will have the potential to receive nexiguran ziclumeran, participants will have the option to cross over to the opposite study arm at Month 12 or Month 18, depending on study criteria.

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
• Amyloidosis, Familial
• Amyloidosis, Hereditary
• Amyloidosis
• Polyneuropathies
• Amyloid Neuropathies
• Amyloid Neuropathies, Familial
• Peripheral Nervous System Disease
• Peripheral Nervous System Diseases
• Metabolism, Inborn Errors
• Metabolic Diseases

Interventions

Type	Name	Description
BIOLOGICAL	nexiguran ziclumeran	nexiguran ziclumeran 55 mg by single IV infusion
DRUG	Normal Saline as Placebo	Normal saline (0.9% NaCl) by single IV infusion

Timeline

• Start date: 2024-11-22
• Primary completion: 2027-07-01
• Completion: 2028-08-01
• First posted: 2024-11-04
• Last updated: 2026-04-16

Locations

14 sites across 6 countries: Argentina, Brazil, Mexico, Singapore, Taiwan, Thailand

Regulatory

• FDA-regulated drug study