Safety and efficacy of leriglitazone in childhood cerebral adrenoleukodystrophy (NEXUS): an interim analysis of an open-label, phase 2/3 trial.

ABSTRACT:

Background

Cerebral adrenoleukodystrophy rapidly progresses in approximately 90% of untreated patients. Current treatment, haematopoietic stem-cell transplantation (HSCT), is associated with high morbidity and is not widely available. Lower risk treatments that can be administered immediately upon lesion identification are needed. Leriglitazone, a peroxisome proliferator-activated receptor gamma agonist, may slow disease progression.

Methods

NEXUS (NCT04528706), a 96-week, phase 2/3, open-label, multicentre study conducted between February 13, 2020 and April 2025, enrolled boys aged 2-12 years with X-linked adrenoleukodystrophy with white matter lesions. Participants received oral leriglitazone once-daily. The primary endpoint is the proportion of patients with arrested dise

SUBMITTER: Garcia-Cazorla A 

PROVIDER: S-EPMC12159931 | biostudies-literature | 2025 Jun

REPOSITORIES: biostudies-literature