Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia

• Status: Recruiting
• Trial end date: 2023-12-07
• Target enrollment: 0
• Participant gender: All

Summary

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals

Criteria

Inclusion Criteria:

• Heterozygous Familial Hypercholesterolemia (HeFH) diagnosed either by genetic testing or on phenotypic criteria

• Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening

• Fasting triglycerides <400 mg/dL (4.5 mmol/L) at screening

• On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for ≥ 30 days before screening

• Estimated glomerular filtration rate (eGFR) >30 mL/min/1.73 m2 at screening

Exclusion Criteria:

• Homozygous familial hypercholesterolemia (HoFH)

• Active liver disease

• Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome

• Major adverse cardiovascular events within 3 months prior to randomization

• Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)

• Recent and/or planned use of other investigational medicinal products or devices

Other protocol-defined inclusion/exclusion criteria may apply