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

Overview

About this study

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

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

- 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 1000
pg/mL (or greater than or equal to 2000 pg/mL if participant has known atrial
fibrillation)

Exclusion Criteria:

- 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 within 6 months prior to study dosing

- Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2

- Liver failure

- Uncontrolled blood pressure

- Unable or unwilling to take vitamin A supplementation for the duration of the study

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Eligibility last updated 12/14/23. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Martha Grogan, M.D.

Open for enrollment

Contact information:

Amyloid Research Team

(507) 266-4426

AMYLOIDTEAMRES@mayo.edu

More information

Publications

Publications are currently not available
.
CLS-20566568

Mayo Clinic Footer