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.
Who can participate
Age range18 Years – 85 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Diagnosis of ATTRv-PN
* Karnofsky Performance Status (KPS) ≥ 60
Exclusion Criteria:
* 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