Last updated 3 days 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.
SponsorIntellia Therapeutics
Study typeInterventional
Conditions
Amyloidosis
Requirements
To 85 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT06672237
