Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous 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 homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C).

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

Details

• Lead Sponsor: Novartis Pharmaceuticals

Criteria

Inclusion Criteria:

• Homozygous Familial Hypercholesterolemia (HoFH) diagnosed by genetic confirmation

• Fasting LDL-C >130 mg/dL (3.4 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:

• Documented evidence of a null (negative) mutation in both LDLR alleles

• Heterozygous familial hypercholesterolemia (HeFH)

• Active liver disease

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

• Major adverse cardiovascular events within 1 month prior to randomization

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

• Treatment with mipomersen or lomitapide (within 5 months of screening)

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

Other protocol-defined inclusion/exclusion criteria may apply