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Promising phase IIb results with sonlicromanol in mitochondrial myopathies caused by the m.3243A>G mutation
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Positive results have been published from a phase IIb clinical trial evaluating the drug candidate sonlicromanol in mitochondrial diseases caused by the m.3243A>G mutationModification soudaine et transmissible du matériel génétique. Elle peut être spontanée ou induite par des agents dits » mutagènes » (radiations, produits toxiques,…).. The trial was conducted in two stages:
- A first, randomised, controlled part in which 27 patients were divided into three groups, depending on whether they received 50 mg, 100 mg sonlicromanol or a placeboLe placebo est un produit dont la présentation est identique à celle d’un médicament, mais qui ne contient pas de principe actif. Dans un essai clinique, un placebo est utilisé pour mesurer l’action réelle du médicament testé, en comparant les effets du médicament testé contenant le principe actif et ceux du placebo. twice a day for 28 days.
- An extension phase, which included 15 of these patients. All received 100 mg of sonlicromanol twice a day for one year.
The results of the first part showed that :
- The primary endpoint of the Cogstate IDN score (a measure of attention) was not achieved.
- Nevertheless, after adjusting for the severity of this criterion at inclusion, a significant improvement compared with placebo was observed in the most severely affected patients.
- The secondary results were analysed using this adjusted model. The results showed an improvement in the HADS-D depression scale, the BDI (Beck Depression Inventory) score and the CFQ (25-item Cognitive Failure Questionnaire) score.
In the extension phase:
- Greater positive changes were seen with long-term treatment.
- Significant improvements were observed particularly in the TAP (Test of Attentional Performance), the BDI, the EQ-5D-5L VAS quality of life scale and pain assessed by the RAND SF-36 score.
- Most patients improved on the Five Times Sit-to-Stand Test.
Although the primary endpoint was not met, several positive results justify the setting up of a phase IIIAu cours d’un essai clinique de phase III, un médicament, pour lequel on a déterminé lors d’essais antérieurs l’innocuité et le dosage optimum (essais de phase I et II), est administré à un grand groupe de malades, sur une longue durée, dans le but d’évaluer son efficacité thérapeutique en la comparant à celle d’un traitement de référence ou un placebo. Il permet aussi de mettre en évidence les interactions indésirables et les effets secondaires du traitement à moyen terme. Au terme de cet essai, le médicament peut obtenir une autorisation de mise sur le marché. trial in patients with diseases due to the m.3243A>G mutation, who are more homogeneous and more severely affected, for 52 weeks, to determine more precisely the potential of this drug candidate, which is also well tolerated.