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 UCSD
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 765 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, Counterfeit Drugs

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 accepting new patients
    San Diego 5391811 California 5332921 92103 United States
  • Cedars-Sinai Medical Center accepting new patients
    Los Angeles 5368361 California 5332921 90048 United States
  • Western Greater Los Angeles VA Medical Center accepting new patients
    Los Angeles 5368361 California 5332921 90073 United States
  • Stanford University accepting new patients
    Palo Alto 5380748 California 5332921 94305 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Intellia Therapeutics
ID
NCT06128629
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 765 study participants
Last Updated