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 Children (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol

Status: Recruiting
Location: See all (53) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 11
Healthy Volunteers: f
View:

• Male or female participants, 6 to \<12 years of age at screening

• HeFH diagnosed either by genetic testing or on phenotypic criteria

• Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening

• For participants 8 to \<12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants \<8 years, the use of background lipid-lowering treatment is based on investigator's discretion.

• Participants on lipid-lowering therapies (such as statin and/or e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation.

Locations
United States
California
UC San Francisco Medical Center
RECRUITING
San Francisco
Washington, D.c.
Children's National Hospital
NOT_YET_RECRUITING
Washington D.c.
Childrens National Hospital
RECRUITING
Washington D.c.
Florida
Excel Medical Clinical Trials LLC
RECRUITING
Boca Raton
New York
Icahn School of Med at Mt Sinai
RECRUITING
New York
Utah
Primary Childrens Medical Center
NOT_YET_RECRUITING
Salt Lake City
West Virginia
West Virginia Childrens Hospital
RECRUITING
Morgantown
Other Locations
Argentina
Novartis Investigative Site
RECRUITING
Caba
Austria
Novartis Investigative Site
RECRUITING
Salzburg
Novartis Investigative Site
RECRUITING
Vienna
Belgium
Novartis Investigative Site
RECRUITING
Brussels
Novartis Investigative Site
RECRUITING
Leuven
Brazil
Novartis Investigative Site
RECRUITING
Fortaleza
Novartis Investigative Site
RECRUITING
Porto Alegre
Novartis Investigative Site
RECRUITING
Rio De Janeiro
Novartis Investigative Site
RECRUITING
São Paulo
China
Novartis Investigative Site
RECRUITING
Beijing
Novartis Investigative Site
RECRUITING
Shanghai
France
Novartis Investigative Site
RECRUITING
Marseille
Novartis Investigative Site
RECRUITING
Nantes
Novartis Investigative Site
RECRUITING
Paris
Germany
Novartis Investigative Site
RECRUITING
Freiburg Im Breisgau
Novartis Investigative Site
RECRUITING
Hanover
Greece
Novartis Investigative Site
RECRUITING
Athens
Novartis Investigative Site
RECRUITING
Ioannina
Hong Kong Special Administrative Region
Novartis Investigative Site
RECRUITING
Hong Kong
Hungary
Novartis Investigative Site
RECRUITING
Budapest
Israel
Novartis Investigative Site
RECRUITING
Jerusalem
Novartis Investigative Site
RECRUITING
Ramat Gan
Italy
Novartis Investigative Site
RECRUITING
Milan
Novartis Investigative Site
RECRUITING
Roma
Novartis Investigative Site
RECRUITING
Torino
Novartis Investigative Site
RECRUITING
Verona
Malaysia
Novartis Investigative Site
RECRUITING
Kuala Lumpur
Netherlands
Novartis Investigative Site
RECRUITING
Amsterdam
Poland
Novartis Investigative Site
RECRUITING
Bialystok
Novartis Investigative Site
RECRUITING
Gdansk
Novartis Investigative Site
RECRUITING
Lodz
Portugal
Novartis Investigative Site
RECRUITING
Lisbon
Novartis Investigative Site
RECRUITING
Porto
South Africa
Novartis Investigative Site
RECRUITING
Bloemfontein
Spain
Novartis Investigative Site
RECRUITING
Barcelona
Novartis Investigative Site
RECRUITING
Cadiz
Novartis Investigative Site
RECRUITING
Elche
Novartis Investigative Site
RECRUITING
Málaga
Novartis Investigative Site
RECRUITING
Pamplona
Novartis Investigative Site
RECRUITING
Seville
Taiwan
Novartis Investigative Site
RECRUITING
Taipei
Novartis Investigative Site
RECRUITING
Taipei
Turkey
Novartis Investigative Site
RECRUITING
Adana
Novartis Investigative Site
RECRUITING
Ankara
Novartis Investigative Site
RECRUITING
Izmir
United Kingdom
Novartis Investigative Site
RECRUITING
West Midlands
Contact Information
Primary
Novartis Pharmaceuticals
novartis.email@novartis.com
1-888-669-6682
Backup
Novartis Pharmaceuticals
+41613241111
Time Frame
Start Date: 2024-12-09
Estimated Completion Date: 2029-04-15
Participants
Target number of participants: 51
Treatments
Experimental: Inclisiran
Year 1 - inclisiran sodium subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium subcutaneous injection (given at Days 450 and 630)
Placebo_comparator: Placebo
Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium subcutaneous injection (given at Days 360, 450, and 630)
Sponsors
Leads: Novartis Pharmaceuticals

This content was sourced from clinicaltrials.gov