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: Nov 24, 2022

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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).

Condition 
Familial Hypercholesterolemia - Heterozygous
Phase 
Phase 3
Overall status 
Recruiting
Start date 
Jan 27, 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 subcutaneous injection)

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

United States
Tucson Medical Center
Recruiting
Tucson, 85712 - Arizona
Contact: (520-324-6290) - [email protected] - Marc Jacobson, MD
United States
Childrens National Hospital
Recruiting
Washington, 20010 - District of Columbia
Contact: (202-476-5000) - [email protected] - Sarah Clauss, MD
United States
Excel Medical Clinical Trials LLC
Recruiting
Boca Raton, 33434 - Florida
Contact: (561-756-8206) - [email protected] - Seth Baum, MD
United States
Washington University School of Medicine
Recruiting
Saint Louis, 63110 - Missouri
Contact: (314-362-8681) - [email protected] - Anne Goldberg, MD
United States
Dartmouth Hitchcock Medical Center
Recruiting
Lebanon, 03756 - New Hampshire
Contact: (603-695-2745) - [email protected] - Marc Hofley, MD
United States
Goryeb Children s Hospital
Recruiting
Morristown, 07960 - New Jersey
Contact: (973-971-4340) - [email protected] - Kanhere Mansi Mukund, MD
United States
Icahn School of Medicine at Mount Sinai
Recruiting
New York, 10029 - New York
Contact: (212-659-9174) - [email protected] - Robert Rosenson, MD
United States
Wake Forest U of Health Sciences
Recruiting
Winston-Salem, 27157 - North Carolina
Contact: Michael Shapiro, MD (336-713-7085) - [email protected] - Michael Shapiro, MD
United States
Cincinnati Children's Hospital Medical Center
Recruiting
Cincinnati, 45229-3039 - Ohio
Contact: (513-636-4744) - [email protected] - Amy Shah, MD
United States
Children's Hospital of Philadelphia
Recruiting
Philadelphia, 19104 - Pennsylvania
Contact: (215-590-1804) - [email protected] - Julie Brothers, MD
United States
Childrens Hospital Pittsburgh of UPMC
Recruiting
Pittsburgh, 15224 - Pennsylvania
Contact: (412-864-8655) - [email protected] - Brenda Mendizabal, MD
United States
University of Tennessee Health Science Center
Recruiting
Memphis, 38163 - Tennessee
Contact: (901-448-4166) - [email protected] - John Jefferies, MD
United States
Primary Children's Medical Center
Recruiting
Salt Lake City, 84113 - Utah
Contact: (801-213-7608) - [email protected] - Adam Ware, MD
United States
West Virginia Children's Hospital
Recruiting
Morgantown, 26505 - West Virginia
Contact: (304-293-1232) - [email protected] - Lee Pyles
United States
American Family Children's Hospital
Recruiting
Madison, 53792 - Wisconsin
Contact: (608-262-5024) - [email protected] - Amy Peterson, MD
United States
Argentina
Novartis Investigative Site
Recruiting
Ciudad Autonoma de Bs As, C1119ACN
Buenos Aires
Argentina
Novartis Investigative Site
Recruiting
Ciudad de Formosa, P3600
Formosa
Argentina
Brazil
Unidade de pesquisa clinica - Hospital Universitario Walter Cantidio
Recruiting
Fortaleza, 60430275 - Ceara
Contact: Diego Lima, Trial Coordinator (+55 85 33668590) - [email protected] -
Brazil
Nucleo de Pesquisa Clinica do Rio Grande do Sul
Recruiting
Porto Alegre, 90430-001 - RS
Contact: Luis Canani, MD (+555137796400) - [email protected] -
Brazil
Novartis Investigative Site
Recruiting
Sao Paulo, 04012-909
SP
Brazil
Setor de Lípides, Aterosclerose e Biologia
Recruiting
Sao Paulo, 04023-900 - SP
Contact: (+55-11-99295-4701) - [email protected] - Maria Izar
Brazil
Heart Institute (InCOr) HCMFUSP
Recruiting
Sao Paulo, 05403 000 - SP
Contact: Raul Santos Filho, Prof. Dr. (+55 11994822460) - [email protected] -
Brazil
Croatia
Novartis Investigative Site
Recruiting
Zagreb, 10000
-
Croatia
Czech Republic
Novartis Investigative Site
Recruiting
Praha 2, 128 08
-
Czech Republic
Novartis Investigative Site
Recruiting
Praha 5, 150 06
-
Czech Republic
France
Novartis Investigative Site
Recruiting
Besancon Cedex, 25030
-
France
Novartis Investigative Site
Recruiting
Bron Cedex, 69677
-
France
Novartis Investigative Site
Recruiting
Toulouse Cedex, 31059
-
France
Germany
Universitaetsmedizin Mannheim
Recruiting
Mannheim, 68305 - Baden-Wuerttemberg
Contact: Claudia Foellinger (+49 (0) 623 832751) - [email protected] -
Germany
KKIM UK Frankfurt/Main
Recruiting
Frankfurt, 60590
Contact: Ulrich Paetow, Dr. Med. (+49 (0) 69630180400)
Germany
Universitaetsklinikum Freiburg
Recruiting
Freiburg, 79106
Contact: (+49 (0) 761 270 43220) - [email protected] - Dagmar Eckert, Trial Coordinator (+49 (0) 761 270 44661) - [email protected] - Karl Otfried Schwab, Prof. Dr.
Germany
Greece
University General Hospital of Ioannina
Recruiting
Ioannina, 455 00 - GR
Contact: Georgia Anastasiou, Dr. (+30 6955516501) - [email protected] -
Greece
Hippokrateion General Hospital of Athens Greece
Recruiting
Athens, 115 27
Contact: Charalambos Vlachopoulos, Professor (+30 6972272727) - [email protected] -
Greece
Metropolitan Hospital
Recruiting
Athens, 18547
Contact: Genovefa Kolovou, Director Cardiologist (+30 210 4807139) - [email protected] -
Greece
Hippokratiou Hospital, Thessaloniki, Greece
Recruiting
Thessaloniki, 54642
Contact: Michael Doumas, Assoc Prof Internal Medicine (+30 2310 892070) - [email protected] -
Greece
Hungary
Novartis Investigative Site
Recruiting
Budapest, 1085
-
Hungary
Israel
Lipid Research
Recruiting
Jerusalem, 9112001
Contact: Shoshi Shpitzen, Trial Coordinator (+9722 6776897) - [email protected] -
Israel
Lipids Center Sheba Medical Center, Israel
Recruiting
Ramat Gan, 52621
Contact: (972 35303492) - [email protected] - Hofit Cohen
Israel
Italy
Novartis Investigative Site
Recruiting
Milano, 20162
MI
Italy
Novartis Investigative Site
Recruiting
Modena, 41124
MO
Italy
Novartis Investigative Site
Recruiting
Roma, 00100
RM
Italy
Novartis Investigative Site
Recruiting
Roma, 00165
RM
Italy
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
Malaysia
UiTM Sungai Buloh
Recruiting
Sungai Buloh, 47000 - Selangor Darul Ehsan
Contact: Fatin Aqilah Abu Yazid (+6019-9700511) - [email protected] -
Malaysia
Novartis Investigative Site
Recruiting
Kuala Lumpur, 59100
-
Malaysia
Netherlands
Novartis Investigative Site
Recruiting
Rotterdam, 3015 GD
Zuid Holland
Netherlands
Novartis Investigative Site
Recruiting
Amsterdam, 1105 AZ
-
Netherlands
Norway
Novartis Investigative Site
Recruiting
Oslo, 0514
-
Norway
Russian Federation
Research Institute for Complex Issues of Cardiovascular Disease
Recruiting
Kemerovo, 650002
Contact: Vasily Kashtalap, Dr. (+73842643153) - [email protected] -
Russian Federation
Institute of Internal Prev. Med.
Recruiting
Novosibirsk, 630090
Contact: Mikhail Voyevoda, Prof. (+7 913 950 50 88) - [email protected] -
Russian Federation
Singapore
KK Women's and Children's Hospital
Recruiting
Singapore, 229899
Contact: Fabian Yap, Dr. (+65 63941127) - [email protected] -
Singapore
Slovakia
Novartis Investigative Site
Recruiting
Bratislava, 833 40
-
Slovakia
Slovenia
University Medical Centre Ljubljana, Div. of Pediatric Dept. of Endocrinology, Diabetes and Metabolic Diseases
Recruiting
Ljubljana, 1000
Contact: Urh Groselj, MD, PhD (+38615229235) - [email protected] -
Slovenia
South Africa
Novartis Investigative Site
Recruiting
Bloemfontein, 9301
Free State
South Africa
Novartis Investigative Site
Recruiting
Pretoria, 0184
Gauteng
South Africa
Novartis Investigative Site
Recruiting
Somerset West, 7130
Western Cape
South Africa
Novartis Investigative Site
Recruiting
Cape Town, 7925
-
South Africa
Spain
Hospital Reina Sofia
Recruiting
Cordoba, 14004 - Andalucia
Contact: Francisco Fuentes Jim&#xE9;nez (957012902) - [email protected] - Francisco Fuentes Jim&#xE9;nez, MD
Spain
Hospital Virgen de la Vcitoria
Recruiting
Malaga, 29010 - Andalucia
Contact: Pedro Valdivielso (639113712) - [email protected] - Miguel Angel Sanchez Chaparro, MD
Spain
Hospital Universitario Virgen del Rocio
Recruiting
Sevilla, 41013 - Andalucia
Contact: Maria Jes&#xFA;s Dom&#xED;nguez (630485685) - [email protected] - Ovidio Mu&#xF1;iz, MD
Spain
Hospital Central de Asturias
Recruiting
Oviedo, 33011 - Asturias
Contact: (686981076) - [email protected] - Isolina Ria&#xF1;o Gal&#xE1;n, MD
Spain
Hospital Clínic de Barcelona
Recruiting
Barcelona, 08036 - Catalunya
Contact: Judith Viaplana (696876589) - [email protected] - Daniel Zamb&#xF3;n Rados, MD
Spain
Hospital Abente y Lago
Recruiting
A Coruna, 15001
Contact: José Luís Díaz Díaz (628 544 729) - [email protected] - José Luís Díaz Díaz, MD
Spain
Taiwan
Far Eastern Memorial Hospital
Recruiting
New Taipei, 22060
Contact: (+886 928 520688) - [email protected] - Jao Shwann Liang, MD
Taiwan
Taipei Veterans General Hospital
Recruiting
Taipei, 11217
Contact: (+886 955 604673) - [email protected] - Min Ji Charng, MD
Taiwan
Thailand
Novartis Investigative Site
Recruiting
Khon Kaen, 40002
THA
Thailand
King Chulalongkorn Memorial Hospital
Recruiting
Bangkok, 10330
Contact: [email protected] - Sirinuch Chomtho, MD, PhD
Thailand
Novartis Investigative Site
Recruiting
Bangkok, 10400
-
Thailand
Turkey
Novartis Investigative Site
Recruiting
Istanbul, 34098
TUR
Turkey
Novartis Investigative Site
Recruiting
Adana,
-
Turkey
Novartis Investigative Site
Recruiting
Ankara, 06490
-
Turkey
Dept. of Pediatric Metabolic Diseases
Recruiting
Ankara, 06500
Contact: Fatih Ezgu, Prof., MD (+90 312 202 6027) - [email protected] -
Turkey
Novartis Investigative Site
Recruiting
Istanbul, 34093
-
Turkey
Novartis Investigative Site
Recruiting
Izmir, 35040
-
Turkey
United Kingdom
Novartis Investigative Site
Recruiting
Bromwich, B71 4HJ
-
United Kingdom
Novartis Investigative Site
Recruiting
Middlesex, UB9 6JH
-
United Kingdom
Vietnam
Novartis Investigative Site
Recruiting
Hanoi, 100000
-
Vietnam

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