This study is in progress, not accepting new patients

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

a study on Cardiomyopathy Hereditary Amyloidosis

Summary

Eligibility
for people ages 18-90 (full criteria)
Location
at San Diego, California and other locations
Dates
study started
study ends around

Description

Summary

To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.

Official Title

MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Details

This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 1200 participants, who will be randomized to receive a single infusion of either NTLA-2001 or placebo.

Keywords

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy, TTR, Amyloidosis, Cardiomyopathy, NTLA-2001, ATTR-CM, Transthyretin, ATTR, TTR-mediated amyloidosis, Amyloidosis, Hereditary, Amyloidosis, Hereditary, Transthyretin-Related Amyloidosis, Transthyretin amyloid cardiomyopathy, TTR cardiomyopathy, Wild-type TTR, V122I, Amyloidosis, Wild Type, Amyloidosis, Hereditary, Transthyretin-Related, Cardiomyopathies, Familial Amyloidosis

Eligibility

You can join if…

Open to people ages 18-90

  • Documented diagnosis of ATTR amyloidosis with cardiomyopathy
  • Medical history of heart failure (HF)
  • Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention
  • Screening NT-proBNP, a blood marker of HF severity, greater than or equal to 600 pg/mL and less than 10,000 pg/mL

You CAN'T join if...

  • New York Heart Association (NYHA) Class IV HF
  • Polyneuropathy Disability score of IV (confined to wheelchair or bed)
  • Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
  • History of active malignancy within 3 years prior to screening
  • RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed
  • Initiation of tafamidis or acoramidis within 56 days prior to study dosing
  • Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m2
  • Liver failure
  • Uncontrolled blood pressure
  • Unable or unwilling to take vitamin A supplementation for the duration of the study

Locations

  • University of California, San Diego (UCSD) - Medical Center
    San Diego California 92103 United States
  • Western Greater Los Angeles VA Medical Center
    Los Angeles California 90073 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Intellia Therapeutics
ID
NCT06128629
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 1200 study participants
Last Updated