Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous 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 Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-13)

ClinicalTrials.gov Identifier: NCT04659863

Novartis Reference Number: CKJX839C12302

Last Update: May 05, 2022

See if you pre-qualify

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 homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C).

Condition 
Familial Hypercholesterolemia - Homozygous
Phase 
Phase 3
Overall status 
Recruiting
Start date 
Feb 16, 2021
Completion date 
Dec 09, 2024
Gender 
All
Age(s)
12 Years - 17 Years (Child)

Interventions

Drug
Inclisiran
Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection
Drug
Placebo
Sterile normal saline (0.9% sodium chloride in water for injection) for subcutaneous injection

Eligibility 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

Study Locations

United States
Tucson Medical Center
Recruiting
Tucson, 85712 - Arizona
Contact: (520-324-6290) - [email protected] - Marc Jacobson
United States
Excel Medical Clinical Trials LLC
Recruiting
Boca Raton, 33434 - Florida
Contact: (561-756-8206) - [email protected] - Seth Baum, MD
United States
Canada
Novartis Investigative Site
Recruiting
Quebec, G1V 4W2
-
Canada
France
Novartis Investigative Site
Recruiting
Bron Cedex, 69677
-
France
Greece
University General Hospital of Ioannina
Recruiting
Ioannina, 455 00 - GR
Contact: Georgia Anastasiou, Dr. (+30 6955516501) - [email protected] -
Greece
Metropolitan Hospital
Recruiting
Athens, 18547
Contact: Genovefa Kolovou, MD (+30 210 4807139) - [email protected] -
Greece
Lebanon
Hotel Dieu de France Hospital
Recruiting
Ashrafieh, 166830
Contact: (00961 (0) 3 406001) - [email protected] - Selim Jambart, Professor
Lebanon
American University of Beirut Medical Center
Recruiting
Beirut, 1107 2020
Contact: (9611350000) - [email protected] - Hala Tfayli, Ass. Prof. of Clin. Pediatrics
Lebanon
Netherlands
Novartis Investigative Site
Recruiting
Amsterdam, 1105 AZ
-
Netherlands
Novartis Investigative Site
Recruiting
Rotterdam, 3015 GD
-
Netherlands
Slovenia
University Medical Centre Ljubljana, Div. of Pediatric Dept. of Endocrinology, Diabetes and Metabolic Diseases
Recruiting
Ljubljana, 1000
Contact: Urh Groselj (+38615229235) - [email protected] -
Slovenia
Switzerland
Novartis Investigative Site
Recruiting
Geneve 14, 1211
-
Switzerland
Turkey
Novartis Investigative Site
Recruiting
Istanbul, 34098
TUR
Turkey
Novartis Investigative Site
Recruiting
Izmir, 35040
-
Turkey

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
Name: 
Novartis Pharmaceuticals
Phone: 

Have a question?

Call 1-888-669-6682 or email [email protected]