A CLINICAL STUDY TO ASSESS THE EFFICACY AND SAFETY OF LERIGLITAZONE IN ADULT MALE SUBJECTS WITH CEREBRAL ADRENOLEUKODYSTROPHY

Cerebral adrenoleukodystrophy (cALD)

The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, whichever comes earlier, in subjects treated with leriglitazone compared to placebo.

Change from Baseline in Loes score., To evaluate the efficacy of leriglitazone compared to placebo on the following clinical parameters: - Time to increase of at least 1 MFD in the NFS-MFD scale - Change from Baseline in ADL – section II of the Friedreich’s Ataxia Rating Scale (FARS) - Time to Major Neurocognitive Impairment – defined as dementia with loss of all intellectual functions requiring constant supervision or assistance, Performed at final analysis (with the analysis following the primary endpoint being met): • Change from Baseline of NFS • Change from Baseline of Symbol Digit Modalities Test (SDMT) cognitive assessment • Clinician Global Impression - Severity and Clinician Global Impression - Change of symptoms (CGI-S, CGI-C, and anchored scale for CGI-C) • Change from Baseline in plasma neurofilament light chain protein (NfL) • Change from Baseline in European Quality of Life 5 Dimensions (EQ-5D-5L), To evaluate the effects of leriglitazone on complementary biomarkers, and clinical and imaging parameters, To assess PK parameters of leriglitazone (plasma) – predicted area under the time-concentration curve (AUC), minimum plasma concentration (Cmin), and maximum plasma concentration (Cmax) (only at the 6-month visit)., Cerebrospinal fluid leriglitazone and M3 concentrations change from Baseline (only at 12-month visit and if optional CSF samples have been collected).

No related papers found yet.

France, Germany, Spain