Last updated 40 hours ago

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

50 patients around the world
Available in Brazil
This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 50 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.
Intellia Therapeutics
50Patients around the world

This study is for people with

Rare diseases
Amyloidosis

Requirements for the patient

To 85 Years
All Gender

Medical requirements

Diagnosis of ATTRv-PN.
Karnofsky Performance Status (KPS) ≥ 60.
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.
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