Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia
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 Heterozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-16)
ClinicalTrials.gov Identifier: NCT04652726
Novartis Reference Number: CKJX839C12301
Last Update: May 03, 2022
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.
Study Description
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).
Interventions
Eligibility 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
Study Locations
Contacts
Have a question?
Call 1-888-669-6682 or email [email protected]