drugset / Trial / NCT03851705
A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
RandomizedParallel-groupDouble-blindTreatment
Summary
This study was a Phase III,A two-part (double-blind placebo-controlled/open-label) multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in subjects with homozygous familial hypercholesterolemia (HoFH).
Timeline
- Start
- 2019-02-06
- Primary completion
- 2020-03-02
- Completion
- 2021-09-09
Outcome
Missed primary endpoint
registry p = 0.9047; Mean Difference (Final Values) -1.68 (95% CI -29.19 to 25.83) NCT03851705 ↗
paper The difference was not statistically significant between the inclisiran and placebo groups. PMID 37850379 ↗
Publications
- Raal F, Durst R, Bi R, Talloczy Z, Maheux P, Lesogor A, Kastelein JJP; ORION-5 Study Investigators. Efficacy, Safety, and Tolerability of Inclisiran in Patients With Homozygous Familial Hypercholesterolemia: Results From the ORION-5 Randomized Clinical Trial. Circulation. 2024 Jan 30;149(5):354-362. doi: 10.1161/CIRCULATIONAHA.122.063460. Epub 2023 Oct 18.
- Warden BA, Duell PB. Inclisiran: A Novel Agent for Lowering Apolipoprotein B-containing Lipoproteins. J Cardiovasc Pharmacol. 2021 Aug 1;78(2):e157-e174. doi: 10.1097/FJC.0000000000001053.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Inclisiran | Small interfering RNA (siRNA) | 300 mg | Subcutaneous |