MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: NTLA-2001, Placebo.
- Who it may be relevant to
- Registry conditions: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy. Basic parameters: 18 years — 90 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Argentina, Australia, Austria, Belgium +21
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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)
Overview
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
Detailed description
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.
Interventions
- Biological NTLA-2001
NTLA-2001 (55mg) by IV infusion - Drug Placebo
Normal saline (0.9% NaCl) by IV infusion
Primary outcome measures
- Composite outcome of cardiovascular (CV) mortality and CV events [Time frame: Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to approximately 5 years]
Secondary outcome measures (2)
- Change in baseline to month 18 in serum TTR [Time frame: Baseline, Month 18]
- Change from baseline to month 18 in KCCQ-OS score [Time frame: Baseline, Month 18]
Eligibility criteria
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 600 pg/mL and less than 10,000 pg/mL
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 or acoramidis within 56 days prior to study dosing
- Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m\^2
- History of liver disease
- Uncontrolled blood pressure
- Unable or unwilling to take vitamin A supplementation for the duration of the study
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 38 centers
- University of Arizona — Tucson
- Cedars-Sinai Medical Center — Los Angeles
- Western Greater Los Angeles VA Medical Center — Los Angeles
- Stanford University — Palo Alto
- University of California, San Diego (UCSD) - Medical Center — San Diego
- University of Colorado Denver — Denver
- Yale University School of Medicine — New Haven
- MedStar Washington Hospital Center - MedStar Heart and Vascular Institute — Washington D.C.
- … and 30 more centers
Brazil · 9 centers
- Centro de Pesquisa NUPEC - Instituto Orizonti em Belo Horizonte — Belo Horizonte
- Complexo Hospitalar de Niterói (CHN-DASA) — Niterói
- CAPED - Centro Avancado de Pesquisa — Ribeirão Preto
- HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto — Ribeirão Preto
- Cardiopulmonar da Bahia S. A. (Hospital Cardio Pulmonar) — Salvador
- Hospital Israelita Albert Einstein — São Paulo
- Instituto Dante Pazzanese de Cardiologia — São Paulo
- Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP — São Paulo
- … and 1 more center
Australia · 8 centers
- The Canberra Hospital — Garran
- Westmead Hospital — Westmead
- Cairns Hospital — Cairns
- Eastern Health - Box Hill Hospital — Box Hill
- Fiona Stanley Hospital — Perth
- Royal Adelaide Hospital — Adelaide
- The Alfred Hospital — Melbourne
- Princess Alexandra Hospital — Woolloongabba
Italy · 7 centers
Center list to be confirmed — check the primary protocol.
United Kingdom · 7 centers
Center list to be confirmed — check the primary protocol.
Argentina · 6 centers
- Fundacion Favalro — Buenos Aires
- Hospital Italiano de Buenos Aires — Buenos Aires
- ICBA — Buenos Aires
- Fundacion Favaloro — Ciudad Autonoma de Buenos Aire
- Hospital Italiano de Buenos Aires — Ciudad Autonoma de Buenos Aire
- Hospital Privado de Rosario — Rosario
Spain · 6 centers
Center list to be confirmed — check the primary protocol.
Belgium · 5 centers
- AZ Sint-Jan Brugge — Bruges
- Universite Libre de Bruxelles (ULB) - Hopital Erasme — Brussels
- Antwerp University Hospital — Edegem
- UZ Leuven — Leuven
- Centre Hospitalier Regional de la Citadelle — Liège
France · 5 centers
- AP-HP Hopital Henri Mondor — Créteil
- AP-HM - Hopital de la Timone — Marseille
- AP-HP Hopital Bichat - Claude Bernard — Paris
- CHU Rennes - Hopital Pontchaillou — Rennes
- CHU de Toulouse - Hopital Rangueil — Toulouse
Canada · 4 centers
- Alberta Health Services - University of Calgary — Calgary
- Vancouver Coastal Health Research Institute — Vancouver
- University Health Network - Toronto General Hospital — Toronto
- McGill University Health Centre — Montreal
Germany · 4 centers
Center list to be confirmed — check the primary protocol.
Japan · 4 centers
Center list to be confirmed — check the primary protocol.
South Korea · 4 centers
Center list to be confirmed — check the primary protocol.
Israel · 3 centers
Center list to be confirmed — check the primary protocol.
Netherlands · 3 centers
Center list to be confirmed — check the primary protocol.
Taiwan · 3 centers
Center list to be confirmed — check the primary protocol.
Austria · 2 centers
- Medizinische Universitaet Innsbruck — Innsbruck
- Universitaesklinikum St. Poelten — Sankt Pölten
Denmark · 2 centers
- Aarhus University Hospital — Aarhus
- Rigshospitalet — Copenhagen
New Zealand · 2 centers
Center list to be confirmed — check the primary protocol.
Portugal · 2 centers
Center list to be confirmed — check the primary protocol.
Singapore · 2 centers
Center list to be confirmed — check the primary protocol.
Sweden · 2 centers
Center list to be confirmed — check the primary protocol.
Czechia · 1 center
- Fakultni nemocnice u sv. Anny v Brne — Brno
Hungary · 1 center
Center list to be confirmed — check the primary protocol.
Mexico · 1 center
Center list to be confirmed — check the primary protocol.
Norway · 1 center
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT06128629 · ITL-2001-CL-301