Safety, Tolerability, and Pharmacokinetics of Oral Ferric Maltol in Children With Iron Deficiency: Phase 1 Study.

UNLABELLED: Iron deficiency is common in children and can have negative effects on behavior and function. Standard oral ferrous iron replacement is poorly absorbed and can cause treatment-limiting gastrointestinal adverse events (AEs). Ferric maltol is formulated to improve gastrointestinal absorption and tolerability versus oral ferrous compounds. In adult phase 3 trials, it increased hemoglobin and iron stores versus placebo, with a gastrointestinal AE profile similar to placebo. Here, we assess different doses of ferric maltol in children with iron deficiency.

METHODS

This phase 1 trial involved children of age 10 to 17 years with ferritin <30 µg/L (or <50 µg/L with transferrin saturation [TSAT] <20%). Children were randomized 1:1:1 to oral ferric maltol 7.8 mg, 16.6 mg, or 30 mg twice daily for 9 days and once on day 10. The primary outcomes were iron uptake measures (serum iron and TSAT) and population pharmacokinetic analyses.

RESULTS

The trial included 37 children (mean age 14.0 years; baseline mean ± standard deviation ferritin 16.3 ± 8.02 µg/L). Ferric maltol increased iron uptake nondose-proportionally: serum iron and TSAT plateaued between the 2 higher doses on day 1 and were comparable across all doses on day 10. Twenty children (54%) experienced AEs (all mild/moderate, gastrointestinal 32%), with similar frequencies in each group.

CONCLUSIONS

All 3 ferric maltol doses increased iron uptake in children with iron deficiency, even over the short study duration, and were well tolerated. Nondose-dependent changes in serum iron and TSAT indicate physiologic regulation of iron uptake to meet the body's needs.