Balance

Study design

Study design

APPROACH study design

The APPROACH trial was a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial that evaluated the efficacy and long-term safety of Waylivra as an adjunct to diet over 52 weeks.1,2

Inclusion criteria1:

  • Confirmed homozygote, compound heterozygote, or double heterozygote for known loss-of-function mutations in FCS-causing genes (such as LPL, APOC2, GPIHBP1, or LMF1) or post-heparin plasma LPL of ≤20% of normal activity
  • Lactescent serum or documentation of fasting TG measurement ≥880 mg/dl

Exclusion criteria:

  • Patients taking Glybera† (alipogene tiparvovec) within 2 years prior to screening were excluded from the study.1

Baseline characteristics2,3

*Patients receive Waylivra 285 mg once weekly, or less frequently based on safety and tolerability.

**<20 grams of fat per day. †Glybera was withdrawn from the market in October 2017.

***Among 14 patients with unidentified genetic mutations, six had mutations in LPL or accessory proteins not annotated or predicted to be inactivating; five had no known relevant mutations. Genetic testing was not performed in three patients because of lack of consent; they were enrolled based on other criteria. APOA5, apolipoprotein A-5; APOC2, apolipoprotein C-II; BMI, body mass index; GPIHBP1, glycosylphosphatidylinositol-anchored high-density lipoprotein-binding protein 1; LMF1, lipase maturation factor 1; LPL, lipoprotein lipase; n, number of patients; SD, standard deviation; TG, triglyceride

References

  1. Waylivra® SmPC.
  2. Blom DJ, et al. J Clin Lipidol. 2018;12:1234–1243
  3. Witztum JL et al. N Engl J Med 2019;381:531–42;