Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol

ReclutandoFase 3ClinicalTrials.gov

ID: NCT06597019RENIS: IS004948Tipo: INTERVENTIONALInicio: 9 de dic de 2024Fin estimado: 15 de abr de 2029

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Resumen

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).

Descripción detallada

This is a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy.

Elegibilidad

Edad mínima: 6 YearsEdad máxima: 11 YearsSexo: ALL

Criterios de inclusión

Male or female participants, 6 to \<12 years of age at screening
HeFH diagnosed either by genetic testing or on phenotypic criteria
Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening
For participants 8 to \<12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants \<8 years, the use of background lipid-lowering treatment is based on investigator's discretion.
Participants on lipid-lowering therapies (such as statin and/or e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation.

Criterios de exclusión

Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
Homozygous familial hypercholesterolemia (HoFH)
Body weight \<16 kg at the screening and/or randomization (Day 1) visit
Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation \>3x ULN, or total bilirubin elevation \>2x ULN (except patients with Gilbert's syndrome)
Pregnant or nursing females
Recent and/or planned use of other investigational medicinal products or devices

Intervenciones

drug

Inclisiran

Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg and inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg. The dose level is based on the participant's body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively.

drug

Placebo

Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Ubicaciones

Novartis Investigative Site

Buenos Aires, Argentina

RECRUITING

Novartis Investigative Site

CABA, Argentina

RECRUITING

Patrocinadores

PrincipalNovartis Pharmaceuticals

Contactos

Novartis Pharmaceuticals

CONTACT

novartis.email@novartis.com 1-888-669-6682

Novartis Pharmaceuticals

CONTACT

+41613241111

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