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.
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.
More information about the study, including its locations and contacts, can be found here.
“We are pleased that enrollment in the FRAMES trial has started,” Marc Martinell, PhD, CEO of Minoryx, said in a press release. “Based on preclinical studies there is a strong rationale for developing leriglitazone in this indication and we are currently exploring a number of additional conditions affecting the central nervous system, where leriglitazone may provide potential benefit for patients.”
In preclinical models of several diseases, it was seen to modulate cellular signals involved in mitochondrial dysfunction (defective energy production), oxidative stress, neuroinflammation, nerve cell degeneration, and demyelination (destruction of the nerve cells’ protective myelin layer).
Results from a Phase 1 trial demonstrated that leriglitazone is well-tolerated and can cross the blood-brain barrier. It can activate PPAR gamma signals within the central nervous system, which suggests that it may hold therapeutic activity for several neurological disorders.
Minoryx is testing the drug in a Phase 2/3 trial (ADVANCE, NCT03231878) in adult patients with adrenomyeloneuropathy, the most common form of X-linked adrenoleukodystrophy (X-ALD).
“I’m delighted to see that Minoryx’s leriglitazone is being assessed in multiple rare CNS diseases, an area where there is a high unmet medical need for novel treatments,” said Alexandra Durr, MD, PhD, principal investigator at the Brain and Spine Institute of La Pitié-Salpêtrière University Hospital (ICM), and coordinator of the FRAMES study.
“I’m looking forward to the completion and a positive outcome of this clinical trial, which could bring a long-awaited treatment option for Friedreich’s ataxia patients,” she added.