A patient with Friedreich’s ataxia has been dosed with Minoryx Therapeutics’ investigational therapy leriglitazone in the ongoing Phase 2 FRAMES trial, the company announced.
This first patient was enrolled at the Hospital La Paz in Madrid, and was administered the new therapy at the end of April by Dr. Francisco Javier Rodríguez de Rivera and his team.
More clinical centers have joined the trial (2018-004405-64 or NCT03917225) in an effort to assess the safety and efficacy of leriglitazone as treatment for patients with Friedreich’s ataxia.
Among them are ICM in Paris, Hospital Sant Joan de Déu in Barcelona, Spain; Hôpital Erasme-ULB in Brussels, and Universitätsklinikum RWTH in Aachen, Germany, which are now recruiting participants.
The trial aims to enroll 36 patients, ages 12 and up, who will be randomized to take either leriglitazone or placebo orally for about 48 weeks.
Researchers will assess, as a primary outcome, changes in patients’ cervical spinal cord area, as determined by magnetic resonance imaging (MRI) measurements, compared to placebo.
They will also evaluate the impact of the investigational therapy on the levels of several biomarkers related to Friedreich’s ataxia, as well as effect on fatigue, ataxia severity, and quality of life.