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)

Activo, no reclutaFase 3ClinicalTrials.gov

ID: NCT06128629Tipo: INTERVENTIONALInicio: 13 de dic de 2023Fin estimado: 1 de abr de 2028

Genética

Traducción no disponible, mostrando original

Resumen

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

Descripción detallada

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.

Elegibilidad

Edad mínima: 18 YearsEdad máxima: 90 YearsSexo: ALL

Criterios de inclusión

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

Criterios de exclusión

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
Liver failure
Uncontrolled blood pressure
Unable or unwilling to take vitamin A supplementation for the duration of the study

Intervenciones

biological

NTLA-2001

NTLA-2001 (55mg) by IV infusion

drug

Placebo

Normal saline (0.9% NaCl) by IV infusion

Ubicaciones

Fundacion Favalro

Buenos Aires, Argentina

Hospital Italiano de Buenos Aires

Buenos Aires, Argentina

ICBA

Buenos Aires, Argentina

Fundacion Favaloro

Ciudad Autonoma de Buenos Aire, Argentina

Hospital Italiano de Buenos Aires

Ciudad Autonoma de Buenos Aire, Argentina

Hospital Privado de Rosario

Rosario, Argentina

Patrocinadores

PrincipalIntellia Therapeutics

ColaboradorRegeneron Pharmaceuticals

Aviso: La información de este ensayo proviene de fuentes públicas y tiene carácter exclusivamente informativo. No constituye asesoramiento médico, regulatorio ni de ningún otro tipo. Las traducciones son automatizadas y no poseen validez oficial. Consultá siempre con un profesional de la salud. Ver Términos de Uso.

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