Estudio de Fase 3, global, doble ciego, aleatorizado y controlado con placebo para evaluar la eficacia y seguridad de ION-682884 en pacientes con miocardiopatía amiloide mediada por transtirretina (ATTR CM)

Activo, no reclutaFase 3ClinicalTrials.gov

ID: NCT04136171Tipo: INTERVENTIONALInicio: 13 de mar de 2020Fin estimado: 1 de ago de 2026

Resumen

Evaluar la eficacia de eplontersen en comparación con placebo en participantes con ATTR-CM que reciben el estándar de atención (SoC) disponible.

Descripción detallada

Este es un estudio multicéntrico, doble ciego, en 1438 participantes, quienes serán aleatorizados para recibir inyecciones subcutáneas (SC) de eplontersen o placebo una vez cada 4 semanas. Los participantes también recibirán dosis suplementarias diarias de la cantidad diaria recomendada de vitamina A.

Elegibilidad

Edad mínima: 18 YearsEdad máxima: 90 YearsSexo: ALL

Criterios de inclusión

Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent. If engaged in sexual relations of child-bearing potential, agree to use 1 highly effective contraceptive method
Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the participant or the participant's non-pregnant female partner must be using a highly effective contraceptive method
Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining OR technetium scintigraphy (99mTc -3,3-diphosphono-1,2- propanodicarboxylic acid \[DPD-Tc\], 99m Tc-pyrophosphate \[PYP-Tc\], or 99m Tc-hydroxymethylene-diphosphonate \[HMDP-Tc\]) with Grade 2 or 3 cardiac uptake in the absence of abnormal light chains ratio, centrally confirmed
End-diastolic interventricular septum thickness of \> 12 millimeters (mm) on Screening echocardiogram
New York Heart Association (NYHA) class I-III

Criterios de exclusión

Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA), coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
Cardiomyopathy not primarily caused by ATTR-CM, for example, cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease
Monoclonal gammopathy of undetermined significance (MGUS) and/or alterations in immunoglobulin free light chain (FLC) ratio unless fat, bone marrow, or heart biopsy confirming the absence of light chain and the presence of TTR protein by mass spectrometry or immunoelectron microscopy. For participants with chronic kidney disease (CKD) and without presence of monoclonal protein in blood and urine, the acceptable FLC ratio is 0.26-2.25. Results different from that may be discussed with local hematologist, Investigator and Medical Monitor if the risks associated with the biopsy outweigh the benefits
Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) or anticipated liver transplant or LVAD within 1 year after randomization
Current or previous treatment with Tegsedi™ (inotersen) or Onpattro™ (patisiran) or other oligonucleotide or ribonucleic acid (RNA) therapeutic (including small interfering ribonucleic acid \[siRNA\]; does not apply to COVID-19 mitochondrial \[mRNA\] vaccinations)
Current treatment with diflunisal, doxycycline, with or without ursodeoxycholic acid, and/or non-dihydropyridine calcium-channel blocker (e.g., verapamil, diltiazem). Participants receiving any of these agents must respect a wash-out period of 14 days before randomization.

Intervenciones

drug

Eplontersen

Eplontersen by subcutaneous injection

drug

Placebo

Eplontersen-matching placebo by subcutaneous injection

Ubicaciones

Instituto Cardiovascular de Buenos Aires

Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina

Hospital Italiano de Buenos Aires

Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina

Patrocinadores

PrincipalIonis Pharmaceuticals, Inc.

ColaboradorAstraZeneca

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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