P4.017: Efficacy And Safety Of A Novel Oral Iron (Ferric Maltol) In Children And Adolescents; A Randomized, Multicenter, Open-Label, Active Comparator Study

Background: Iron deficiency anemia (IDA) is treatable, but limited by tolerance issues with current oral iron preparations. Ferric maltol is a novel non-salt oral iron designed to overcome limitations of ferrous based preparations. It is a stable ferric iron and tri-maltol complex, approved for the use in adults with ID/IDA , demonstrating proven efficacy and tolerability.
This phase 3 study (NCT05126901; 21/YH/0200) compared ferric maltol to ferrous sulfate across 23 sites (US, Puerto Rico, UK).

Methods: In a 12-week open-label study, 61 children/adolescents (2-17 years) with IDA were randomized 1:1 to oral ferric maltol or ferrous sulfate using computer-randomized permutation. Children 2-11 years received 15mg iron as ferric maltol suspension BID, 12-17 years received 30 mg BID, and the ferrous sulfate group received 3 mg/kg/dose BID. Safety was assessed by AEs; efficacy by Hb, iron, TSAT, and ferritin at Week 12. The children rated palatability on a 5-point Facial Hedonic Scale; younger children were evaluated by parents/clinicians. The study was stopped early after a planned interim analysis showed a statistically significant Hb change from baseline in children (N=33) receiving ferric maltol at Week 12 [2-sided (100-3.45%) CI; p< 0.0001].

Results: Primary endpoints were descriptive statistical comparisons of (1) Hb change from baseline and (2) safety and gastrointestinal tolerability between ferric maltol and ferrous sulfate groups. A clinically significant Hb increase from baseline to Week 12 was observed in both groups: 12.5 g/L increase in in the ferric maltol group vs. 11.5 g/L increase in the ferrous sulfate group (mITT population).
Nine patients experienced AEs in both groups (29%/30%, respectively) (all mild/moderate), with more patients reporting drug related AEs in the ferrous sulfate group (4 patients, 6 AEs) than the ferric maltol group (2 patients, 10 AEs). One child in the 2-17 age group withdrew from the ferrous sulfate group due to AEs (gastrointestinal disorder, abdominal pain, nausea); none in the ferric maltol group.
Iron increase was higher in the ferrous sulfate group at week 4. By Week 12, the ferric maltol group had the highest mean iron increase (5.77 µmol/L vs. ferrous sulfate 3.64 µmol/L). TSAT changes at Week 12 were similar for the ferric maltol and ferrous sulfate groups (7.7% vs. 6.5%), while ferritin rose most in the ferrous sulfate group.
Among age 6-17, ferrous sulfate had more negative taste ratings (34.6% very bad) than ferric maltol (6.9% very bad). Ferric maltol was rated better for smell, swallowing, and ease of intake than ferrous sulfate.

Conclusion: Ferric maltol significantly improves Hb levels and iron storage within 12 weeks while providing a well-tolerated preferential taste alternative to ferrous sulfate in children and adolescents at the administered dosages.