Resumen

Este estudio de Fase 3 evaluará la seguridad y eficacia de la inyección de plozasiran (ARO-APOC3) en participantes adultos con hipertrigliceridemia grave (HTGS). Tras la firma del consentimiento informado, los participantes elegibles serán aleatorizados para recibir 4 dosis (una cada 3 meses) de plozasiran o placebo, y serán evaluados en cuanto a eficacia y seguridad. Tras el mes 12, se ofrecerá a los participantes elegibles la oportunidad de continuar en una extensión abierta opcional bajo un protocolo separado.

Elegibilidad

Edad mínima: 18 YearsSexo: ALL

Criterios de inclusión

Established diagnosis of severe hypertriglyceridemia (SHTG) and prior documented evidence (medical history) of fasting TG levels of ≥ 500 mg/dL (≥5.65mmol/L)
Mean fasting TG level ≥500 mg/dL (≥5.65 mmol/L) collected at 2 separate and consecutive visits at least 7 days apart and no more than 17 days apart during the screening period
Fasting low density lipoprotein-cholesterol (LDL-C) ≤130 mg/dL (≤3.37 mmol/L) at screening
Screening HbA1C ≤9.0%
Willing to follow diet counseling and maintain a stable low-fat diet
Males, or nonpregnant (who do not plan to become pregnant) nonlactating females, who are ≥18 years of age at screening
Must be on standard of care lipid and TG lowering medications per local guidelines (unless documented as intolerant as determined by the Investigator, including an inability to safely administer or re-administer a specific drug because of fear, preference, genetic, clinical, or metabolic considerations, or due to a previous adverse reaction associated with, attributed to, or caused by specific drug)

Criterios de exclusión

Use of any hepatocyte-targeted small interfering ribonucleic acid (siRNA) that targets lipids and/or triglycerides within 365 days before Day 1 (except inclisiran, which is permitted). Administration of investigational drug and inclisiran must be separated by at least 4 weeks
Use of any other hepatocyte-targeted siRNA or antisense oligonucleotide molecule within 60 days or within 5-half-lives before Day 1 based on plasma pharmacokinetics (PK), whichever is longer
Known diagnosis of familial chylomicronemia syndrome (FCS) (type 1 Hyperlipoproteinemia) by documentation of confirmed homozygote or double heterozygote for loss-of-function mutations in type 1-causing genes
Acute pancreatitis within 4 weeks prior to screening
Body mass index \>45kg/m\^2

Ubicaciones

Research Center 8

Ramos Mejía, Buenos Aires, Argentina

Research Center 10

San Isidro, Buenos Aires, Argentina

Research Center 7

Temperley, Buenos Aires, Argentina

Research Center 4

Córdoba, Córdoba Province, Argentina

Research Center 9

Córdoba, Córdoba Province, Argentina

Research Center 5

Córdoba, Córdoba Province, Argentina

Research Center 3

Córdoba, Córdoba Province, Argentina

Research Center 6

Buenos Aires, Argentina

Research Center 2

Buenos Aires, Argentina

Research Center 1

Buenos Aires, Argentina

Estudio de Fase 3, Doble Ciego, Controlado con Placebo para Evaluar la Eficacia y Seguridad de plozasiran en Adultos con Hipertrigliceridemia Grave

Resumen

Elegibilidad

Criterios de inclusión

Criterios de exclusión

Intervenciones

Plozasiran Injection

Placebo

Ubicaciones

Research Center 8

Research Center 10

Research Center 7

Research Center 4

Research Center 9

Research Center 5

Research Center 3

Research Center 6

Research Center 2

Research Center 1

Patrocinadores