Who is sponsoring NCT04659863?

NCT04659863 is sponsored by Novartis Pharmaceuticals.

What drugs are being tested in NCT04659863?

Interventions in NCT04659863: Inclisiran, Placebo.

What condition does NCT04659863 study?

NCT04659863 studies Familial Hypercholesterolemia - Homozygous.

Is NCT04659863 still recruiting?

NCT04659863 is currently completed. Last updated 13 January 2026.

Are results published for NCT04659863?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2026-01-13 · How we verify

NCT04659863: ORION-13

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia

Completed Phase 3 Results posted Last updated 13 January 2026

What this trial tests

Phase 3 trial testing Inclisiran in Familial Hypercholesterolemia - Homozygous in 13 participants. Completed in 18 November 2024.

Timeline

16 February 2021

Primary endpoint
30 October 2023

18 November 2024

Quick facts

Lead sponsor	Novartis Pharmaceuticals
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	13
Start date	16 February 2021
Primary completion	30 October 2023
Estimated completion	18 November 2024
Sites	11 locations across Greece, Netherlands, Lebanon, United States, Canada, France, Turkey (Türkiye), Malaysia

Drugs / interventions tested

Inclisiran (INCLISIRAN) — full drug profile →
Placebo

Conditions studied

Familial Hypercholesterolemia - Homozygous — all drugs for Familial Hypercholesterolemia - Homozygous →

Sponsor

Novartis Pharmaceuticals — full company profile →

Who can join

Adults 12 to 17, any sex, with Familial Hypercholesterolemia - Homozygous. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Inclisiran: First Approval.
Lamb YN. · · 2021 · cited 257× · PMID 33620677 · DOI 10.1007/s40265-021-01473-6
Therapeutic siRNA: State-of-the-Art and Future Perspectives.
Friedrich M, Aigner A. · · 2022 · cited 256× · PMID 35997897 · DOI 10.1007/s40259-022-00549-3
RNAi-Based Therapeutics and Novel RNA Bioengineering Technologies.
Traber GM, Yu AM. · · 2023 · cited 184× · PMID 35680378 · DOI 10.1124/jpet.122.001234
Targeting proprotein convertase subtilisin/kexin type 9 (PCSK9): from bench to bedside.
Bao X, Liang Y, Chang H, Cai T, et al · · 2024 · cited 101× · PMID 38185721 · DOI 10.1038/s41392-023-01690-3
Advantages and Disadvantages of Inclisiran: A Small Interfering Ribonucleic Acid Molecule Targeting PCSK9-A Narrative Review.
Merćep I, Friščić N, Strikić D, Reiner Ž. · · 2022 · cited 71× · PMID 35237348 · DOI 10.1155/2022/8129513
Inclisiran, Low-Density Lipoprotein Cholesterol and Lipoprotein (a).
Katsiki N, Vrablik M, Banach M, Gouni-Berthold I. · · 2023 · cited 50× · PMID 37111334 · DOI 10.3390/ph16040577
Clinical Applications of Short Non-Coding RNA-Based Therapies in the Era of Precision Medicine.
Smith ES, Whitty E, Yoo B, Moore A, et al · · 2022 · cited 48× · PMID 35326738 · DOI 10.3390/cancers14061588
Clinical potential of inclisiran for patients with a high risk of atherosclerotic cardiovascular disease.
Nishikido T. · · 2023 · cited 28× · PMID 36717882 · DOI 10.1186/s12933-023-01752-4

Verify or expand the search:

Other trials of Inclisiran

Trials testing the same drug.

NCT07102628 — Evaluation of Efficacy and Safety of Early in Hospital Initiation of Inclisiran Treatment in Patients With Acute Coronar · Phase 3 · recruiting
NCT06770543 — Leqvio rPMS (Regulatory Post-Marketing Surveillance in Korea) · recruiting
NCT06958315 — Inclisiran (Leqvio®) in Patients With Atherosclerotic Vascular Disease and/or Heterozygous Familial Hypercholesterolemia · recruiting
NCT06813911 — Lp(a) Lowering Study of Pelacarsen (TQJ230) With Background Inclisiran in Participants With Elevated Lp(a) and Establish · Phase 3 · recruiting
NCT06858332 — Lipoprotein(a) Levels in Patients With Atherosclerotic Cardiovascular Diseases in Russia · recruiting

Other recruiting trials for Familial Hypercholesterolemia - Homozygous

Currently open trials in the same condition.

NCT06597006 — Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterole · Phase 3 · recruiting
NCT04370899 — Early Detection of Familial Hypercholesterolemia in Children · recruiting

Other Novartis Pharmaceuticals trials

Trials by the same sponsor.

NCT07498335 — Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Atrasentan in Pediatric Patients With Primary · Phase 3 · not yet recruiting
NCT07489573 — Study of Efficacy and Safety of Secukinumab in Chinese Adult Patients With Moderate to Severe Hidradenitis Suppurativa · Phase 4 · not yet recruiting
NCT07484269 — PULSE Registry: for Patients Receiving Lutetium (177Lu) Vipivotide Tetraxetan · not yet recruiting
NCT07416162 — A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy · not yet recruiting
NCT07387926 — Safety and Efficacy of Asciminib in Pediatrics and Young Adults With Relapse/Refractory (r/r) Philadelphia Positive (Ph+ · Phase 1, PHASE2 · not yet recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04659863 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 9 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 13 January 2026

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04659863.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing