- Among children ages 2-5 years with cystic fibrosis (CF) who are homozygous for F508del-CFTR mutation, lumacaftor and ivacaftor appear safe and well-tolerated.
- Pancreatic biomarkers improved while on the therapy.
- Extension study is ongoing.
Why this matters
- These CF transmembrane conductance regulator (CFTR) protein modulators have not previously been studied in children this young.
- Drug and metabolite plasma concentrations were within expected ranges.
- Mean changes, 24 weeks vs baseline:
- Sweat chloride: −31.7 (95% CI, −35.7 to −27.6) mmol/L.
- Fecal elastase concentration: 52.6 (95% CI, 22.5-82.7) μg/g; 3 children achieved normal range.
- Serum immunoreactive trypsinogen (IRT): −130.2 (95% CI, −192.3 to −68.1) ng/mL.
- No new safety signals.
- Phase 3, open-label, 2-part, multicenter study of children ages 2-5 years with confirmed CF caused by homozygous F508del-CFTR mutation.
- Participants received weight-based doses of lumacaftor and ivacaftor every 12 hours, participating in either:
- Part A: pharmacokinetics and safety study, 15 days (n=12); or
- Part B: pharmacodynamics, efficacy, pharmacokinetics, and safety study, 24 weeks (n=60).
- Outcomes: pharmacokinetics and safety.
- Funding: Vertex Pharmaceuticals Incorporated.
- Very small study without a control group.