The CALYX trial is a fully recruited pivotal phase 3, multicenter, randomized (1:1), double-blind, placebo-controlled, clinical study that has been designed to compare the efficacy and safety of leriglitazone with that of placebo in the treatment of cerebral adrenoleukodystrophy (cALD).

NEXUS is a completed 96-week, pivotal, open-label, multicenter study (NEXUS; NCT04528706) of once-daily oral leriglitazone in pediatric patients with cALD. NEXUS enrolled 23 patients and the evaluable population consisted of those patients treated for a minimum of 24 weeks (n=20).

The TREE trial is a fully recruited phase 2a, double-blind and placebo-controlled clinical study that has been designed to assess the safety and tolerability of leriglitazone in paediatric patients with Rett Syndrome (RTT).

The ADVANCE trial is a completed pivotal phase 2/3 randomized, double-blind, placebo-controlled, clinical study with an open-label extension designed to assess the efficacy and safety of leriglitazone in adult male patients with X-linked Adrenoleukodystrophy (X-ALD).

The FRAMES trial is a completed phase 2 randomized, double-blind, placebo-controlled, clinical study that was designed to assess the efficacy and safety of leriglitazone in patients with Friedreich's ataxia (FRDA) with input from EU and US clinical experts and patient advocacy groups.