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A phase III double-blind, placebo-controlled, randomized withdrawal trial of 5-aminolevulinic acid hydrochloride with sodium ferrous citrate for efficacy and safety in patients diagnosed as Leigh syndrome

2025-09-02 endocrinology Title + abstract only
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ObjectiveA phase III, double-blind, placebo-controlled, randomized withdrawal trial of SPP-004 (5-aminolevulinic acid hydrochloride and sodium ferrous citrate) in patients diagnosed as Leigh syndrome (LS) was conducted to confirm the efficacy and safety of SPP-004 in patients with LS showing central nervous system disorders. MethodsFifty-four patients entered a 24-week open-label period of SPP-004 administration. Among them, 28 patients showing improved scores on the Newcastle Paediatric Mitoch...

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