Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH)
NCT ID: NCT03397121
Last Updated: 2020-10-28
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE3
482 participants
INTERVENTIONAL
2017-11-28
2019-09-17
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Inclisiran
Inclisiran sodium 300 milligrams (mg) will be administered as a SC injection on Day 1, Day 90 then every 6 months.
Inclisiran
Inclisiran is a small interfering ribonucleic acid (RNA) that inhibits PCSK9 synthesis.
Placebo
Placebo will be administered as SC injections of saline solution on Day 1, Day 90 then every 6 months.
Placebo
Placebo will be supplied as sterile normal saline (0.9% sodium chloride in water for injection).
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Inclisiran
Inclisiran is a small interfering ribonucleic acid (RNA) that inhibits PCSK9 synthesis.
Placebo
Placebo will be supplied as sterile normal saline (0.9% sodium chloride in water for injection).
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
2. History of HeFH with a diagnosis of HeFH by genetic testing; and/or a documented history of untreated LDL-C of \>190 mg/dL, and a family history of familial hypercholesterolemia, elevated cholesterol or early heart disease that may indicate familial hypercholesterolemia.
3. Serum LDL-C ≥2.6 millimoles (mmol)/liter (L) (≥100 mg/dL) at screening.
4. Fasting triglyceride \<4.52 mmol/L (\<400 mg/dL) at screening.
5. Participants on statins should be receiving a maximally tolerated dose.
6. Participants not receiving statins must have documented evidence of intolerance to all doses of at least 2 different statins.
7. Participants on lipid-lowering therapies (such as a statin and/or ezetimibe) should be on a stable dose for ≥30 days before screening with no planned medication or dose change during study participation.
Exclusion Criteria
2. Uncontrolled cardiac arrhythmia
3. Uncontrolled severe hypertension
4. Active liver disease
5. Females who are pregnant or nursing, or who are of childbearing potential and unwilling to use at least 2 methods of highly effective contraception (failure rate less than 1% per year) (combined oral contraceptives, barrier methods, approved contraceptive implant, long-term injectable contraception, or intrauterine device) for the entire duration of the study. Exemptions from this criterion:
1. Women \>2 years postmenopausal (defined as 1 year or longer since last menstrual period) AND more than 55 years of age.
2. Postmenopausal women (as defined above) and less than 55 years of age with a negative pregnancy test within 24 hours of randomization.
3. Women who are surgically sterilized at least 3 months prior to enrollment.
6. Males who are unwilling to use an acceptable method of birth control during the entire study period (condom with spermicide).
7. Treatment with other investigational products or devices within 30 days or 5 half-lives of the screening visit, whichever is longer.
8. Treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9.
The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
The Medicines Company
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Frederick J. Raal, MD
Role: PRINCIPAL_INVESTIGATOR
University of Witwatersrand, South Africa
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Site 90001-005
Mission Viejo, California, United States
Site 90001-001
Newport Beach, California, United States
Site 90001-015
Stanford, California, United States
Site 90001-047
Boca Raton, Florida, United States
Site 90001-004
Boston, Massachusetts, United States
Site 90001-056
Saint Paul, Minnesota, United States
Site 90001-012
Butte, Montana, United States
Site 90001-112
Las Vegas, Nevada, United States
Site 90001-014
Summit, New Jersey, United States
Site 90001-002
Cincinnati, Ohio, United States
Site 90011-005
Chicoutimi, Quebec, Canada
Site 90011-001
Montreal, Quebec, Canada
Site 90011-002
Québec, Quebec, Canada
Site 90420-001
Prague, , Czechia
Site 90420-006
Prague, , Czechia
Site 90420-005
Trutnov, , Czechia
Site 90045-001
Aalborg, , Denmark
Site 90045-004
Esbjerg, , Denmark
Site 90045-003
Herning, , Denmark
Site 90045-006
Hvidovre, , Denmark
Site 90045-002
Roskilde, , Denmark
Site 90045-005
Viborg, , Denmark
Site 90031-001
Amersfoort, , Netherlands
Site 90031-003
Amsterdam, , Netherlands
Site 90031-009
Hoorn, , Netherlands
Site 90031-006
Tilburg, , Netherlands
Site 90031-005
Utrecht, , Netherlands
Site 90027-004
Cape Town, Western Cape, South Africa
Site 90027-003
Bloemfontein, , South Africa
Site 90027-005
Cape Town, , South Africa
Site 90027-001
Cape Town, , South Africa
Site 90027-008
Cape Town, , South Africa
Site 90027-010
Johannesburg, , South Africa
Site 90027-007
Pretoria, , South Africa
Site 90027-006
Pretoria, , South Africa
Site 90027-009
Witbank, , South Africa
Site 90034-003
A Coruña, , Spain
Site 90034-005
Barcelona, , Spain
Site 90034-004
Córdoba, , Spain
Site 90034-006
L'Hospitalet de Llobregat, , Spain
Site 90034-001
Reus, , Spain
Site 90034-002
Zaragoza, , Spain
Site 90046-002
Gothenburg, , Sweden
Site 90046-001
Stockholm, , Sweden
Site 90046-003
Stockholm, , Sweden
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Dutta S, Shah R, Singhal S, Singh S, Piparva K, Katoch CDS. A systematic review and meta-analysis of tolerability, cardiac safety and efficacy of inclisiran for the therapy of hyperlipidemic patients. Expert Opin Drug Saf. 2024 Feb;23(2):187-198. doi: 10.1080/14740338.2023.2293201. Epub 2023 Dec 19.
Raal FJ, Kallend D, Ray KK, Turner T, Koenig W, Wright RS, Wijngaard PLJ, Curcio D, Jaros MJ, Leiter LA, Kastelein JJP; ORION-9 Investigators. Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia. N Engl J Med. 2020 Apr 16;382(16):1520-1530. doi: 10.1056/NEJMoa1913805. Epub 2020 Mar 18.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2017-002472-30
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
MDCO-PCS-17-03
Identifier Type: -
Identifier Source: org_study_id