APOLLO-B: estudio de fase 3, aleatorizado, doble ciego, controlado con placebo, multicéntrico, para evaluar la eficacia y seguridad de patisirán en pacientes con amiloidosis por transtiretina con miocardiopatía (amiloidosis ATTR con miocardiopatía)

Activo, no reclutaFase 3ClinicalTrials.gov

ID: NCT03997383Tipo: INTERVENTIONALInicio: 12 de sept de 2019Fin estimado: 15 de mar de 2027

Resumen

El propósito de este estudio es evaluar la eficacia y seguridad de patisirán en participantes con amiloidosis ATTR con miocardiopatía.

Elegibilidad

Edad mínima: 18 YearsEdad máxima: 85 YearsSexo: ALL

Criterios de inclusión

Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hereditary ATTR amyloidosis with cardiomyopathy or wild-type ATTR amyloidosis with cardiomyopathy
Medical history of heart failure with at least 1 prior hospitalization for heart failure, or current clinical evidence (signs and symptoms of heart failure)
Clinically stable with no cardiovascular related hospitalizations within 6 weeks of study start
Has never taken tafamidis before (tafamidis naïve) or currently on tafamidis for ≥6 months with evidence of disease progression while on tafamidis treatment
Able to complete ≥150 m on the 6-minute walk test
Screening N-terminal pro B-type natriuretic peptide (NT-proBNP), a blood marker of heart failure severity, \>300 ng/L and \<8500 ng/L; in participants with permanent or persistent atrial fibrillation, screening NT-proBNP\> 600 ng/L and \<8500 ng/L

Criterios de exclusión

Known primary amyloidosis (AL) or leptomeningeal amyloidosis.
Received prior TTR lowering treatment
New York Heart Association heart failure classification of III and at high risk
New York Heart Association heart failure classification of IV
Neuropathy requiring cane or stick to walk, or is wheelchair bound
Estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m\^2
Abnormal liver function
Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
Has non-amyloid disease that significantly affects ability to walk (e.g., severe chronic obstructive pulmonary disease, severe arthritis, or peripheral vascular disease affecting ambulation)
Prior or planned heart, liver, or other organ transplant
Other cardiomyopathy not related to ATTR amyloidosis

Intervenciones

drug

Placebo

Normal saline (0.9% NaCl) matching volume of patisiran doses will be administered intravenously.

drug

Patisiran

Patisiran will be administered by intravenous (IV) infusion.

Ubicaciones

Clinical Trial Site

Buenos Aires, Buenos Aires, Argentina

Clinical Trial Site

San Juan Bautista, Buenos Aires, Argentina

Clinical Trial Site

Córdoba, Córdoba Province, Argentina

Clinical Trial Site

Rosario, Santa Fe Province, Argentina

Clinical Trial Site

Rosario, Santa Fe Province, Argentina

Clinical Trial Site

Rosario, Santa Fe Province, Argentina

Clinical Trial Site

Santa Fe, Santa Fe Province, Argentina

Clinical Trial Site

Buenos Aires, Argentina

Patrocinadores

PrincipalAlnylam Pharmaceuticals

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