NCT04659863CompletedPhase 3
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous 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 Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-13)
Source: ClinicalTrials.gov · Last updated 13 Jan 2026
Brief summary (registry, in English)
This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C).
- Status (registry)
- Completed
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 13 (actual)
- First submitted
- 2 Dec 2020
- Start date
- 16 Feb 2021
- Primary completion date
- 30 Oct 2023
- Lead sponsor
- Novartis Pharmaceuticals
- Diseases
- Familial hypercholesterolaemia
- NCT number
- NCT04659863
- EudraCT number
- 2020-002755-38
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 13
- From 16 Feb 2021 to the primary completion date, 30 Oct 2023
- 11 sites in 8 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
- Treatment with mipomersen or lomitapide (within 5 months of screening)
Other illness
- Active liver disease
- Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
Other
- Documented evidence of a null (negative) mutation in both LDLR alleles
- Heterozygous familial hypercholesterolemia (HeFH)
- Recent and/or planned use of other investigational medicinal products or devices
Who the study is looking for
- Homozygous Familial Hypercholesterolemia (HoFH) diagnosed by genetic confirmation
- Fasting LDL-C \>130 mg/dL (3.4 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
- Male or female participants \>=12 to \<18 years of age at screening
Age in the registry: 12 years to 17 years · Sex: All · Does not accept healthy volunteers
Sites (11)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| - Coordinator of this site? Confirm its status | Québec | Canada | Unknown status |
| - Coordinator of this site? Confirm its status | Bron | France | Unknown status |
| Metropolitan Hospital Coordinator of this site? Confirm its status | Athens | Greece | Unknown status |
| University General Hospital of Ioannina Coordinator of this site? Confirm its status | Ioannina | Greece | Unknown status |
| American University of Beirut Medical Center Coordinator of this site? Confirm its status | Beirut | Lebanon | Unknown status |
| Hotel Dieu de France Hospital Coordinator of this site? Confirm its status | El Achrafiyé | Lebanon | Unknown status |
| - Coordinator of this site? Confirm its status | Kuala Lumpur | Malaysia | Unknown status |
| - Coordinator of this site? Confirm its status | Amsterdam | Netherlands | Unknown status |
| Excel Medical Clinical Trials, LLC Coordinator of this site? Confirm its status | Boca Raton | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Istanbul | Turkey (Türkiye) | Unknown status |
| - Coordinator of this site? Confirm its status | Izmir | Turkey (Türkiye) | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.