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Looking for participantsPhase3

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

Sponsor: Khondrion BV

NCT ID: NCT06451757

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Sonlicromanol (drug), Placebo (drug)
How long the study runs
Study runs about 29 months (dates as stated)
About the drug or intervention
Sonlicromanol — drug: Administration of 90 mg sonlicromanol (100 mg sonlicromanol.HCl) twice daily during 52 weeks · Placebo — drug: Administration of 100 mg placebo twice daily during 52 weeks
Patient visit burden
Not specified by the sponsor

In plain English

This study, run by Khondrion BV, is testing a medicine called sonlicromadol—spelled sonlicromanol in the registry—for its effectiveness and safety in adults with primary mitochondrial diseases. It focuses on people with a specific genetic change called the m.3243A>G mutation, which can cause conditions such as maternally inherited diabetes and deafness (MIDD) and MELAS (mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes).

Who can take part

  • Adults aged 18 or over with a multi-system primary mitochondrial disease
  • A confirmed m.3243A>G mutation with a heteroplasmy level of 20% or more, measured in blood, urine, cheek swab or muscle before joining
  • Chronic fatigue lasting at least 3 months that is not explained by another cause, recorded in medical files and shown on a screening questionnaire (Neuro-QoL fatigue score above 22)
  • Muscle weakness (mitochondrial myopathy), shown by being able to complete a 'sit to stand' test 5 times within 30 seconds, taking at least 11 seconds
  • Signed informed consent
  • Other inclusion rules apply as set out in the study protocol

Who may not be able to

  • Taking another investigational medicine within the last 3 months, or planning to during the study
  • Bone problems, movement problems or long-term ulcers that could affect the sit-to-stand test
  • Stomach or bowel surgery, or severe digestive problems, that could stop the medicine being absorbed properly
  • Significant lung disease or heart disease
  • Certain heart procedures within the last 3 months
  • A heart rhythm measure (QTcF) above 450 milliseconds in men or 470 milliseconds in women
  • Certain structural heart problems, heart failure (class II or above), or poorly controlled heart conditions
  • Family history of unexplained fainting, certain inherited heart rhythm syndromes, or sudden death before age 60
  • Certain heart rhythm problems on an ECG (heart tracing), such as atrial fibrillation or higher-grade heart block
  • Acute heart failure within the last 3 months
  • Other exclusion rules apply as set out in the study protocol

What taking part involves

  • • Taking sonlicromanol, the study medicine being tested for effectiveness and safety
  • • Not stated — ask the trial team

Time commitment: Not stated — ask the trial team about how long the study lasts, how many visits are needed and what taking part involves.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
18 Years and over
Who
All
Number of participants
220
Started
2026-04-14
Last checked
2026-06

Plain English Summary

What is this study?

  • • Testing a new treatment for mitochondrial diseases
  • • Phase3 - 220 participants
  • • 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

Who can take part?

  • • Ages 18 Years and over
  • • Diagnosed with mitochondrial diseases

Where?

  • • London - University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

More detail

The KHENERFIN study is investigating the medicine sonlicromanol. The study aims to see if sonlicromanol can reduce symptoms of fatigue and reduce the impact of fatigue on daily life. The study also investigates if sonlicromanol improves physical abilities like balance control and lower limb skeletal muscle strength in people with mitochondrial disease. In addition to these primary objectives, the study evaluates the efficacy of sonlicromanol on selected secondary and exploratory outcomes. It also assesses 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. Participants cannot change their assigned rreatment. During the screening period, which lasts a maximum of 4 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. Sonlicromanol will be supplied in tablet form, containing 90 mg of sonlicromanol (equivalent to 100 mg of sonlicromanol.HCl), with the tablets embossed accordingly or provided as a placebo. The study medication must be taken twice daily during the treatment period of 52 weeks. Up to 220 subjects with a confirmed mitochondrial DNA tRNALeu(UUR) 3243A\>G mutation will be randomly assigned in a 1:1 ratio to receive either sonlicromanol or placebo.

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

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What we know so far

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 18 Years and over
  • Who can join: All genders

Biomarkers mentioned

PR

What the study is looking for

  • ✓Inclusion criteria
  • ✓Signed agreement to take part
  • ✓Males and females aged ≥18 years with a multi-system primary mitochondrial disease.
  • ✓Presence of chronic fatigue (not attributable to other etiologies than PMD):
  • ✓Patient self-reported chronic fatigue for at least 3 months prior to the Screening Visit and recorded in the...
See the full criteria
Inclusion criteria 1. Signed Informed Consent 2. Males and females aged ≥18 years with a multi-system primary mitochondrial disease. 3. A confirmed mitochondrial DNA tRNALeu(UUR) m.3243A\>G mutation (m.3243A\>G PMD) plus an age adjusted heteroplasmy percentage ≥ 20% in white blood cells \[=blood heteroplasmy/0.977(age+12)\]. Or in urine (urinary epithelial cells), or buccal smear or skeletal muscle (results (obtained per local guidance) ≥ 20% must be available prior to the subject being randomized). 4. Presence of chronic fatigue (not attributable to other etiologies than PMD): 1. Patient self-reported chronic fatigue for at least 3 months prior to the Screening Visit and recorded in the clinical patient files; AND 2. Presence of fatigue (raw total score \>22), assessed by Neuro-QoL SFv1-F at Screening. 5. Presence of mitochondrial myopathy defined as: 5xSST at Screening and Baseline should be ≥ 11 seconds and participant must demonstrate the ability to complete the test at baseline (i.e., complete the test within 30 seconds). 6\. Other Inclusion criteria per protocol. Exclusion criteria 1. Treatment with any IMP within 3 months (or 5 times the half-life of the IMP, whichever is longer) prior to screening or plans to use an IMP (other than the study intervention) during the study. 2. Bone deformities, motor abnormalities or chronic ulcers that in the opinion of the PI may interfere with and/or confound the interpretation of the subject's performance during the 5 times sit to stand test (5XSST). 3. Surgery of gastrointestinal tract that might interfere with drug absorption. Or severe GI dysmotility, chronic vomiting, diarrhea, bouts of pseudo-obstruction which will impair appropriate IMP absorption in the opinion of the investigator. 4. Clinically significant respiratory disease and/or cardiac disease (medical history or current clinical findings) in the opinion of the investigator. 5. Prior interventional cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to screening. 6. QTcF \> 450 msec (men) or QTcF \> 470 msec (women). 7. Structural heart disease based on cardiac MRI or Echocardiography (e.g., clinically significant valve disease; i.e., aortic or mitral valve stenosis or regurgitation) and/or abnormal conduction (QRS \>120 msec, PR \< 120 msec), and/or repolarization (QTcF \> 450 msec (men) or QTcF \> 470 msec (women)). Myocardial function (LVEF \<52% in men and \< 54% in women), symptomatic ischemic heart disease (inducible ischemia or coronary obstruction), and/or pathologic hypertrophy (e.g. \> 15mm septal or posterior wall thickness), that is not well controlled under current specialized care. Subjects with congestive heart failure class II and above should also be excluded. 8. Family history of unexplained/uninvestigated syncope or congenital long and short QT syndrome or sudden death (under the age of 60). ECG evidence of acute or recent ischemia, acute or Recent Myocardial Infraction, atrial fibrillation, high grade AV Blocks (Second Degree AV Block Type II or Third-degree AV Block), complete Heart Block or active conduction system abnormalities with the exception of any of the following: 1. First degree atrioventricular (AV)-block 2. Second degree AV-block Type 1 (Mobitz Type 1/Wenckebach type) 3. Right bundle branch block. 9. History of acute heart failure (within the last 3 months). 10. Higher degree of AV-blocks (AVB II° or III°). 11. Other exclusion criteria per protocol

Where Is This Study? (1 UK site)

University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery

London WC1N 3BG, United Kingdom

Recruiting
Site contact (verified)
Robbert Pitceathly, Dr.Principal Investigator

How to Get in Touch

Jasper Levink, MSc.

Sponsor contact

CONTACT

+31 24 7635000 Khenerfin@khondrion.com

G. Ruiterkamp, MSc.

Sponsor contact

CONTACT

+31 24 7635000 Khenerfin@khondrion.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-06