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

NCT06451757 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 220

Last updated 2026-01-29

No results posted yet for this study

Summary

The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of people like balance control and lower limb skeletal muscle strength in people with mitochondrial disease.

In this study, the effects of sonlicromanol are compared against a placebo, a tablet identical in appearance and taste but without the active drug. Participants take either sonlicromanol or placebo twice daily for a treatment duration of 52 weeks.

In addition to these primary objectives, the study evaluates the efficacy of sonlicromanol on secondary and exploratory outcomes, as well as its safety and tolerability after one year of treatment.

Conditions

  • Mitochondrial Diseases
  • Maternally Inherited Diabetes and Deafness (MIDD)
  • Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
  • Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation

Interventions

DRUG

Sonlicromanol

Administration of 90 mg sonlicromanol (100 mg sonlicromanol.HCl) twice daily during 52 weeks

DRUG

Placebo

Administration of 100 mg placebo twice daily during 52 weeks

Sponsors & Collaborators

  • Khondrion BV

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-02-28
Primary Completion
2028-09-30
Completion
2028-09-30
FDA Drug
Yes

Countries

  • United States
  • Denmark
  • France
  • Germany
  • Italy
  • Netherlands
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06451757 on ClinicalTrials.gov