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

Launched by INTELLIA THERAPEUTICS · Nov 1, 2024

Keywords

ClinConnect Summary

This clinical trial is testing a new treatment called NTLA-2001 for people with a condition known as ATTRv-PN, which is a type of nerve disorder caused by the buildup of abnormal proteins in the body. The study aims to find out if a single dose of NTLA-2001 is effective and safe compared to a placebo (a treatment that has no active ingredients) in helping individuals with this condition. The trial is currently recruiting participants aged between 65 and 74, and it is open to all genders.

To be eligible for this trial, participants must have a confirmed diagnosis of ATTRv-PN and a good level of overall health, as indicated by a Karnofsky Performance Status score of 60 or higher (which means they can perform some daily activities). However, individuals with certain other health conditions, such as diabetes, severe heart problems, or liver failure, will not be able to participate. If you join the study, you will receive either the treatment or a placebo and will be monitored for any effects. It’s also important to note that participants will need to take vitamin A supplements during the study. This trial could provide valuable information about a potential new option for managing ATTRv-PN.

Gender

ALL

Eligibility criteria

• 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