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 Adolescents (12 to Less Than 18 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-13)

CKJX839C12302

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).

2021-02-16

2023-10-30

2024-12-02

Active (not recruiting)

Early phase I

13

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Parallel (Year 1) to single-group (Year 2)', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'DOUBLE', 'maskingDescription': 'Masked (Year 1) to No Masking (Year 2)', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}}, 'enrollmentInfo': {'count': 13, 'type': 'ACTUAL'}}

2024-03-22