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)
Intellia Therapeutics
60 participants
Nov 22, 2024
INTERVENTIONAL
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.
Eligibility
Inclusion Criteria2
- Diagnosis of ATTRv-PN
- Karnofsky Performance Status (KPS) ≥ 60
Exclusion Criteria10
- 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
- Liver failure
- Hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
- Prior receipt of a TTR silencer (Small interfering RNA (siRNA) or Antisense oligonucleotides (ASOs))
- Estimated Glomerular Filtration Rate < 30 mL/min/1.73 m2
- Unable or unwilling to take vitamin A supplementation for the duration of the study
- History of liver disease
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Interventions
nexiguran ziclumeran 55 mg by single IV infusion
Normal saline (0.9% NaCl) by single IV infusion
Locations(14)
View Full Details on ClinicalTrials.gov
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NCT06672237