Actively Recruiting
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
Led by Novartis Pharmaceuticals · Updated on 2026-02-20
9
Participants Needed
18
Research Sites
215 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).
CONDITIONS
Official Title
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female participants, 2 to less than 12 years of age at screening
- Diagnosed with homozygous familial hypercholesterolemia (HoFH) confirmed by genetic testing
- Fasting LDL cholesterol greater than 130 mg/dL at screening
- On an optimal dose of statin unless statin intolerant, with or without other lipid-lowering therapy
- Stable dose of lipid-lowering therapies for at least 30 days before screening with no planned changes during the study
- Participants on LDL-apheresis for at least 3 months before screening may continue with stable settings during the study
You will not qualify if you...
- Documented null mutations in both LDLR alleles
- Previous treatment with PCSK9 monoclonal antibodies within 90 days before screening
- History of poor response to PCSK9 monoclonal antibody therapy
- Treatment with mipomersen or lomitapide within 5 months before screening
- Secondary hypercholesterolemia such as hypothyroidism or nephrotic syndrome
- Heterozygous familial hypercholesterolemia
- Body weight below 16 kg for ages 6 to under 12 years or below 11 kg for ages 2 to under 6 years
- Active liver disease or unexplained elevated liver enzymes above specified limits
- Pregnant or nursing females
- Recent or planned use of other investigational drugs or devices
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 18 locations
1
UC San Francisco Medical Center
San Francisco, California, United States, 94143
Actively Recruiting
2
UC San Francisco Medical Center
San Francisco, California, United States, 94143
Actively Recruiting
3
Childrens National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
4
Washington Univ School Of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
5
Novartis Investigative Site
Vienna, Austria, 1090
Actively Recruiting
6
Novartis Investigative Site
Beijing, Beijing Municipality, China, 100013
Actively Recruiting
7
Novartis Investigative Site
Frankfurt am Main, Hesse, Germany, 60590
Actively Recruiting
8
Novartis Investigative Site
Ioannina, Greece, 455 00
Actively Recruiting
9
Novartis Investigative Site
Thessaloniki, Greece, 546 42
Actively Recruiting
10
Novartis Investigative Site
Kota Bharu, Kelantan, Malaysia, 16150
Actively Recruiting
11
Novartis Investigative Site
Amsterdam, North Holland, Netherlands, 1105 AZ
Actively Recruiting
12
Novartis Investigative Site
Bloemfontein, Free State, South Africa, 9301
Actively Recruiting
13
Novartis Investigative Site
Taichung, Taiwan, 407219
Actively Recruiting
14
Novartis Investigative Site
Taipei, Taiwan, 111045
Actively Recruiting
15
Novartis Investigative Site
Adana, Saricam, Turkey (Türkiye), 01330
Actively Recruiting
16
Novartis Investigative Site
Ankara, Yenimahalle, Turkey (Türkiye), 06500
Actively Recruiting
17
Novartis Investigative Site
Izmir, Turkey (Türkiye), 35100
Actively Recruiting
18
Novartis Investigative Site
Southampton, United Kingdom, SO16 6YD
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
CONTACT
N
Novartis Pharmaceuticals
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here