ACTIVE_NOT_RECRUITING

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

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Conditions

Transthyretin Amyloidosis (ATTR) With CardiomyopathyTTRAmyloidosisCardiomyopathyNTLA-2001ATTR-CMTransthyretinATTRTTR-mediated amyloidosisAmyloidosis, HereditaryAmyloidosis, Hereditary, Transthyretin-Related AmyloidosisTransthyretin amyloid cardiomyopathyTTR cardiomyopathyWild-type TTRV122IAmyloidosis, Wild Type

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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)

Quick Facts

Study Start:2023-12-13
Study Completion:2028-04
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT06128629

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 90 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Documented diagnosis of ATTR amyloidosis with cardiomyopathy
  2. * Medical history of heart failure (HF)
  3. * Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention
  4. * Screening NT-proBNP, a blood marker of HF severity, greater than or equal to 600 pg/mL and less than 10,000 pg/mL
  1. * New York Heart Association (NYHA) Class IV HF
  2. * Polyneuropathy Disability score of IV (confined to wheelchair or bed)
  3. * Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
  4. * History of active malignancy within 3 years prior to screening
  5. * RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed
  6. * Initiation of tafamidis or acoramidis within 56 days prior to study dosing
  7. * Estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m\^2
  8. * Liver failure
  9. * Uncontrolled blood pressure
  10. * Unable or unwilling to take vitamin A supplementation for the duration of the study

Contacts and Locations

Study Locations (Sites)

University of Arizona
Tucson, Arizona, 85724
United States
Cedars-Sinai Medical Center
Los Angeles, California, 90048
United States
Western Greater Los Angeles VA Medical Center
Los Angeles, California, 90073
United States
Stanford University
Palo Alto, California, 94305
United States
University of California, San Diego (UCSD) - Medical Center
San Diego, California, 92103
United States
University of Colorado Denver
Denver, Colorado, 80045
United States
Yale University School of Medicine
New Haven, Connecticut, 06510
United States
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington D.C., District of Columbia, 20010
United States
Mayo Clinic - Jacksonville
Jacksonville, Florida, 32224
United States
Cleveland Clinic of Florida
Weston, Florida, 33331
United States
Emory University School of Medicine
Atlanta, Georgia, 30322
United States
Northwestern University
Chicago, Illinois, 60611
United States
Indiana University Health
Indianapolis, Indiana, 46202
United States
University of Kentucky Gill Heart Institute
Lexington, Kentucky, 40536
United States
Johns Hopkins University
Baltimore, Maryland, 21287
United States
Brigham and Women's Hospital
Boston, Massachusetts, 02115
United States
Boston University School of Medicine
Boston, Massachusetts, 02118
United States
University of Michigan
Ann Arbor, Michigan, 48109
United States
Mayo Clinic
Rochester, Minnesota, 55905
United States
St. Luke's Hospital
Kansas City, Missouri, 64111
United States
New York University (NYU) School of Medicine - Langone Medical Center
New York, New York, 10016
United States
Columbia University Medical Center
New York, New York, 10032
United States
Duke University Medical Center
Durham, North Carolina, 27710
United States
Cleveland Clinic
Cleveland, Ohio, 44195
United States
Ohio Health Research Institute
Columbus, Ohio, 43214
United States
Oregon Health and Science University (OHSU)
Portland, Oregon, 97239
United States
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, 19104
United States
Allegheny General Hospital
Pittsburgh, Pennsylvania, 15212
United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15219
United States
Medical University of South Carolina
Charleston, South Carolina, 29425
United States
Baylor Scott & White Research Institute
Dallas, Texas, 75201
United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
United States
Baylor College of Medicine
Houston, Texas, 77030
United States
Houston Methodist Hospital
Houston, Texas, 77030
United States
Baylor Scott and White - The Heart Hospital Baylor Plano
Plano, Texas, 75093
United States
INOVA Fairfax Hospital
Falls Church, Virginia, 22042
United States
Virginia Commonwealth University - Department of Internal Medicine
Richmond, Virginia, 23298
United States

Collaborators and Investigators

Sponsor: Intellia Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-12-13
Study Completion Date2028-04

Study Record Updates

Study Start Date2023-12-13
Study Completion Date2028-04

Terms related to this study

Keywords Provided by Researchers

  • 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

Additional Relevant MeSH Terms

  • Transthyretin Amyloidosis (ATTR) With Cardiomyopathy