PATIENTS

The ADVANCE Clinical Study for AMN

The ADVANCE trial is a phase 2/3 randomized, double-blind, placebo-controlled, clinical study with an open-label extension that has been designed to assess the efficacy and safety of MIN-102 as a treatment for adrenomyeloneuropathy (AMN) in male patients with X-linked adrenoleukodystrophy.

The study is divided into two parts. The first part is a blinded placebo-controlled part with a 2:1 (active:placebo) randomization ratio and consists of two years of treatment where different efficacy and safety parameters are evaluated. This first part is followed-up by an open-label extension part where all participants completing part 1 will be eligible to receive MIN-102. This potentially pivotal study has been designed with input from both EMA and FDA as well as EU and US clinical experts in X-ALD and patient advocacy groups.

Over 100 patients are taking part in the ADVANCE study, which is anticipated to provide results by the end of 2020.

For further information visit: https://clinicaltrials.gov/ct2/show/NCT03231878?term=NCT03231878&rank=1

The FRAMES Clinical Study for FRDA

The FRAMES trial is a phase 2 randomized, double-blind, placebo-controlled, clinical study that has been designed to assess the efficacy and safety of leriglitazone as a treatment in patients with Friedreich ataxia (FRDA).

This study has been designed with input from EU and US clinical experts and patient advocacy groups.

Over 30 patients are taking part in the FRAMES study, which is anticipated to provide results by the end of 2020. Recruitment into this study has closed.

For further information visit: https://clinicaltrials.gov/ct2/show/NCT03917225?term=MIN102&rank=2

The NEXUS Clinical Study for cALD

The NEXUS clinical trial is a phase 2, open-label clinical study that has been designed to assess the efficacy and safety of leriglitazone in male pediatric patients with early stage cerebral X-linked adrenoleukodystrophy (cALD).

The NEXUS study recruits cALD patients with early cerebral MRI lesions. All patients will be receiving leriglitazone and they will be carefully monitored for safety, clinical symptoms and changes in cerebral MRI lesions. The study has been designed with input from both EMA and FDA as well as EU and US clinical experts in X-ALD and patient advocacy groups. The study design does not interfere with or delay any standard-of-care interventions.

For further information visit: https://www.clinicaltrialsregister.eu/ctr-search/search?query=2019-000654-59

List of Locations

  • Spain: Hospital Sant Joan de Déu – Barcelona
  • Germany: University Medical Center Hamburg-Eppendorf – Hamburg