The study intends to enrol adult male X-ALD patients with gadolinium-enhancing brain lesions. Credit: sfam_photo /Shutterstock.com.

The US Food and Drug Administration (FDA) has granted clearance to Minoryx Therapeutics to initiate a Phase III clinical trial of leriglitazone to treat adult male X-linked adrenoleukodystrophy (X-ALD) patients with cerebral adrenoleukodystrophy (cALD).

The Phase III CALYX trial will be carried out across selected centres of excellence in South America and the US.

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Dr Ali Fatemi at the Kennedy Krieger Institute in Baltimore will act as the global principal investigator for the study.

CALYX intends to enrol 40 adult male X-ALD patients with gadolinium-enhancing brain lesions and who have refused to undergo haematopoietic stem cell transplantation (HSCT).

Patients will be randomised into a 1:1 ratio to either receive leriglitazone or a placebo.

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The trial includes an adaptive duration, with an initial efficacy read at 18 months.

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It also has efficacy assessments at 27 and 36 months, with an option to complete the trial at any of the three time points upon reaching statistical significance.

Time to death or bedridden with permanent ventilatory support is the primary endpoint of the study.

Measuring radiological progression through the Loes Score is the key secondary endpoint while major functional disability and biomarkers in plasma, such as neurofilament light chain, are the clinical endpoints.

Minoryx CEO Marc Martinell said: “CALYX will be funded from the Series C financing, together with proceeds from our European strategic collaboration with Neuraxpharm.

“We look forward to initiating this trial, which could provide an important therapeutic option for patients suffering from this devastating orphan disease with a major unmet medical need.”

Minoryx intends to start enrolling patients by the end of the second quarter of this year, with results expected by late 2025.