drugset / Trial / NCT04568434
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
RandomizedParallel-groupDouble-blindTreatment
Summary
The purpose of the study was to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
Timeline
- Start
- 2020-11-18
- Primary completion
- 2023-07-14
- Completion
- 2023-10-17
Outcome
Mixed primary results
registry analysis (superiority test); Placebo vs Olezarsen 80 mg; p =0.0009; LS mean difference -43.5 (95% CI -69.085 to -17.921) NCT04568434 ↗
registry analysis (superiority test); Placebo vs Olezarsen 50 mg; p =0.0775; Least squares (LS) mean difference -22.37 (95% CI -47.200 to 2.463) NCT04568434 ↗
Publications
- Results Stroes ESG, Alexander VJ, Karwatowska-Prokopczuk E, Hegele RA, Arca M, Ballantyne CM, Soran H, Prohaska TA, Xia S, Ginsberg HN, Witztum JL, Tsimikas S; Balance Investigators. Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome. N Engl J Med. 2024 May 16;390(19):1781-1792. doi: 10.1056/NEJMoa2400201. Epub 2024 Apr 7.
- Sikora Kessler A, Vera-Llonch M, Karwatowska-Prokopczuk E, Alexander VJ, Ann Q, Mejia Herrera C, Paparrodopoulos S, Xia S, Stroes ESG, Baum SJ, Tsimikas S; Balance Investigators. Olezarsen reduces all-cause health services utilization and improves the treatment experience of patients with familial chylomicronemia syndrome. J Clin Lipidol. 2026 Feb;20(2):299-309. doi: 10.1016/j.jacl.2025.12.018. Epub 2025 Dec 19.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Olezarsen | Antisense oligonucleotide | 50 mg | Subcutaneous |
| Subject | Olezarsen | Antisense oligonucleotide | 80 mg | Subcutaneous |