Actively Recruiting
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
Led by Novartis Pharmaceuticals · Updated on 2025-11-21
51
Participants Needed
66
Research Sites
226 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 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).
CONDITIONS
Official Title
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female participants, 6 to less than 12 years of age at screening
- Diagnosed with heterozygous familial hypercholesterolemia (HeFH) by genetic testing or phenotypic criteria
- Fasting LDL cholesterol greater than 130 mg/dL at screening
- For participants aged 8 to less than 12 years, on an optimal dose of statin unless statin intolerant, with or without other lipid-lowering therapy
- For participants under 8 years, background lipid-lowering treatment use is at investigator's discretion
- Stable dose of lipid-lowering therapy for at least 30 days before screening with no planned changes during the study
You will not qualify if you...
- Previous treatment within 90 days before screening with monoclonal antibodies targeting PCSK9
- Secondary hypercholesterolemia such as hypothyroidism or nephrotic syndrome
- Homozygous familial hypercholesterolemia (HoFH)
- Body weight less than 16 kg at screening or randomization
- Active liver disease or unexplained elevated liver enzymes beyond 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: 66 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
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Not Yet Recruiting
4
Childrens National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
5
Excel Medical Clinical Trials LLC
Boca Raton, Florida, United States, 33434
Actively Recruiting
6
Icahn School of Med at Mt Sinai
New York, New York, United States, 10029
Actively Recruiting
7
Primary Childrens Medical Center
Salt Lake City, Utah, United States, 84113
Not Yet Recruiting
8
Primary Childrens Medical Center
Salt Lake City, Utah, United States, 84113
Actively Recruiting
9
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Actively Recruiting
10
West Virginia Childrens Hospital
Morgantown, West Virginia, United States, 26506
Actively Recruiting
11
West Virginia Childrens Hospital
Morgantown, West Virginia, United States, 26506
Actively Recruiting
12
Novartis Investigative Site
Buenos Aires, Argentina, C1245AAM
Actively Recruiting
13
Novartis Investigative Site
CABA, Argentina, C1181ACH
Actively Recruiting
14
Novartis Investigative Site
Salzburg, Austria, 5020
Actively Recruiting
15
Novartis Investigative Site
Vienna, Austria, 1090
Actively Recruiting
16
Novartis Investigative Site
Brussels, Belgium, 1200
Actively Recruiting
17
Novartis Investigative Site
Leuven, Belgium, 3000
Actively Recruiting
18
Novartis Investigative Site
Fortaleza, Ceará, Brazil, 60430-275
Actively Recruiting
19
Novartis Investigative Site
Rio de Janeiro, Rio de Janeiro, Brazil, 20211-340
Actively Recruiting
20
Novartis Investigative Site
Porto Alegre, Rio Grande do Sul, Brazil, 90020-020
Actively Recruiting
21
Novartis Investigative Site
São Paulo, São Paulo, Brazil, 05403 000
Actively Recruiting
22
Novartis Investigative Site
Beijing, Beijing Municipality, China, 100013
Actively Recruiting
23
Novartis Investigative Site
Shanghai, China, 200127
Actively Recruiting
24
Novartis Investigative Site
Prague, Czechia, 128 08
Actively Recruiting
25
Novartis Investigative Site
Prague, Czechia, 150 06
Actively Recruiting
26
Novartis Investigative Site
Marseille, France, 13885
Actively Recruiting
27
Novartis Investigative Site
Nantes, France, 44093
Actively Recruiting
28
Novartis Investigative Site
Paris, France, 75012
Actively Recruiting
29
Novartis Investigative Site
Freiburg im Breisgau, Baden-Wurttemberg, Germany, 79106
Actively Recruiting
30
Novartis Investigative Site
Frankfurt am Main, Hesse, Germany, 60590
Actively Recruiting
31
Novartis Investigative Site
Hanover, Germany, 30173
Actively Recruiting
32
Novartis Investigative Site
Athens, Greece, 115 27
Actively Recruiting
33
Novartis Investigative Site
Ioannina, Greece, 455 00
Actively Recruiting
34
Novartis Investigative Site
Hong Kong, Hong Kong, 999077
Actively Recruiting
35
Novartis Investigative Site
Budapest, Hungary, 1026
Actively Recruiting
36
Novartis Investigative Site
Jerusalem, Israel, 9112001
Actively Recruiting
37
Novartis Investigative Site
Ramat Gan, Israel, 5265601
Actively Recruiting
38
Novartis Investigative Site
Milan, MI, Italy, 20162
Actively Recruiting
39
Novartis Investigative Site
Roma, RM, Italy, 00165
Actively Recruiting
40
Novartis Investigative Site
Torino, TO, Italy, 10126
Actively Recruiting
41
Novartis Investigative Site
Verona, VR, Italy, 3712
Actively Recruiting
42
Novartis Investigative Site
Kuala Lumpur, Kuala Lumpur, Malaysia, 50586
Actively Recruiting
43
Novartis Investigative Site
Amsterdam, North Holland, Netherlands, 1105 AZ
Actively Recruiting
44
Novartis Investigative Site
Bialystok, Poland, 15-274
Actively Recruiting
45
Novartis Investigative Site
Gdansk, Poland, 80 952
Actively Recruiting
46
Novartis Investigative Site
Lodz, Łódź Voivodeship, Poland, 93-338
Actively Recruiting
47
Novartis Investigative Site
Coimbra, Portugal, 3000-602
Actively Recruiting
48
Novartis Investigative Site
Lisbon, Portugal, 1649-035
Actively Recruiting
49
Novartis Investigative Site
Porto, Portugal, 4050-651
Actively Recruiting
50
Novartis Investigative Site
Porto, Portugal, 4200 319
Actively Recruiting
51
Novartis Investigative Site
Bloemfontein, Free State, South Africa, 9301
Actively Recruiting
52
Novartis Investigative Site
Elche, Alicante, Spain, 03203
Actively Recruiting
53
Novartis Investigative Site
Cadiz, Andalusia, Spain, 11009
Actively Recruiting
54
Novartis Investigative Site
Esplugues, Barcelona, Spain, 08950
Actively Recruiting
55
Novartis Investigative Site
Badalona, Catalonia, Spain, 08916
Actively Recruiting
56
Novartis Investigative Site
Pamplona, Navarre, Spain, 31008
Actively Recruiting
57
Novartis Investigative Site
Barcelona, Spain, 08041
Actively Recruiting
58
Novartis Investigative Site
Málaga, Spain, 29011
Actively Recruiting
59
Novartis Investigative Site
Seville, Spain, 41013
Actively Recruiting
60
Novartis Investigative Site
Taipei, Taiwan, 111045
Actively Recruiting
61
Novartis Investigative Site
Taipei, Taiwan, 11217
Actively Recruiting
62
Novartis Investigative Site
Adana, Saricam, Turkey (Türkiye), 01330
Actively Recruiting
63
Novartis Investigative Site
Ankara, Yenimahalle, Turkey (Türkiye), 06500
Actively Recruiting
64
Novartis Investigative Site
Izmir, Turkey (Türkiye), 35100
Actively Recruiting
65
Novartis Investigative Site
West Midlands, Birmingham, United Kingdom, B4 6NH
Actively Recruiting
66
Novartis Investigative Site
London, United Kingdom, NW3 2QG
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