A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients

Summary

Randomized, double-blind, placebo-controlled study on the effects of MIN-102 on Biochemical, Imaging, neurophysiological, and clinical markers in patients with Friedreich's Ataxia

Timeline

Start

2019-03-26

Primary completion

2020-08-13

Completion

2020-09-14

Outcome

Missed primary endpoint

paper The primary endpoint of change in spinal cord area was not met. PMID 36524101 ↗

release “FRAMES trial data shows promising proof of concept as a therapy for Friedreich's ataxia, despite the inconclusive results of the primary endpoint” minoryx.com ↗

release “Although the primary endpoint was not met in this study, results from secondary endpoints provide evidence for clinical proof of concept for use of leriglitazone in patients with Friedreich’s Ataxia” minoryx.com ↗

Publications

• Pandolfo M, Reetz K, Darling A, Rodriguez de Rivera FJ, Henry PG, Joers J, Lenglet C, Adanyeguh I, Deelchand D, Mochel F, Pousset F, Pascual S, Van den Eede D, Martin-Ugarte I, Vila-Brau A, Mantilla A, Pascual M, Martinell M, Meya U, Durr A. Efficacy and Safety of Leriglitazone in Patients With Friedreich Ataxia: A Phase 2 Double-Blind, Randomized Controlled Trial (FRAMES). Neurol Genet. 2022 Nov 1;8(6):e200034. doi: 10.1212/NXG.0000000000200034. eCollection 2022 Dec.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Leriglitazone	Small molecule	15 mg/ml	Oral

Indications