KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases — KH176-301  

Mitochondrial Diseases Clinical Trial

Official title: A Phase III, Randomised, Double-blind, Placebo-controlled, Parallel-group, Pivotal Trial to Assess the Efficacy and Safety of Sonlicromanol in Adult Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G Variant

Status Not yet recruiting

Clinical Trial Summary

The KHENERFIN study is investigating whether the study medicine, sonlicromanol, is able to improve symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able improve physical abilities of people living with mitochondrial disease, such as balance control and lower limb skeletal muscle strength.

For this study, the effects of sonlicromanol are compared with those from a placebo (study medication that looks like the actual study medicine but contains no active medicine). The study medicine (or placebo) is a powder that is dissolved in water and must be taken twice daily during the treatment period of 52 weeks.

Additionally, the study evaluates the efficacy of sonlicromanol on selected secondary and exploratory outcome measures, as well as the safety and tolerability of sonlicromanol after 52 weeks of treatment with sonlicromanol.

Clinical Trial Description

The KHENERFIN study is investigating whether the study medicine, sonlicromanol, is able to improve symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able improve physical abilities of people living with mitochondrial disease such as balance control and lower limb skeletal muscle strength. Additionally, the study evaluates the efficacy of sonlicromanol on selected secondary and exploratory outcome measures, as well as the safety and tolerability of sonlicromanol. This study is a placebo controlled, double blind study; the effects of sonlicromanol will be compared with a placebo (study medication that looks like the actual study medicine but contains no active medicine). Neither the participants nor the study team know who is receiving the study medicine or placebo and participants are not able to change which treatment they are assigned.

During the screening period, which lasts a maximum of 6 weeks, it is assessed whether the potential participant meets all requirements to participate in the study. Patients who complete the screening phase and are enrolled in the study are randomly (by chance) assigned to receive either the study medicine sonlicromanol or placebo (no active medication). Participants have an equal chance of receiving either sonlicromanol or a placebo. A final follow-up visit is scheduled 2 weeks after taking the last dose of study medication. Total study duration is approximately 60 weeks. The study medicine (as well as placebo) is a powder that is dissolved in water and must be taken twice daily during the treatment period of 52 weeks. A total of 150 patients with a confirmed mitochondrial DNA tRNALeu(UUR) 3243A>G mutation will participate in this study. ;

Related Conditions & MeSH terms

• Acidosis
• Acidosis, Lactic
• Deafness
• Diabetes Mellitus, Type 2
• Hearing Loss
• Maternally Inherited Diabetes and Deafness (MIDD)
• MELAS Syndrome
• Mitochondrial Diseases
• Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
• Mitochondrial Encephalomyopathies

• NCT number: NCT06451757
• Study type: Interventional
• Source: Khondrion BV
• Contact: Jan Smeitink, MD, PhD, MAE
• Phone: +31 24 7635000
• Email: smeitink@khondrion.com
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: January 2, 2025
• Completion date: July 2, 2027