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Tests treatment safety and results for Mitochondrial Diseases
Official title KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
ClinicalTrials.gov ID: NCT06451757
What this study is testing
What is Sonlicromanol?
Sonlicromanol is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for mitochondrial diseases.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- Time commitment: about 52 weeks
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Signed Informed Consent
- Males and females aged ≥18 years with a multi-system primary mitochondrial disease.
- A confirmed mitochondrial DNA tRNALeu(UUR) m.3243A\>G mutation (m.3243A\>G PMD) plus an age adjusted heteroplasmy percentage ≥ 20% in white blood...
- 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 clinical patient files; AND
You likely can't join if
- 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...
- Bone deformities, motor abnormalities or chronic ulcers that in the opinion of the PI may interfere with and/or confound the interpretation of the...
- Surgery of gastrointestinal tract that might interfere with drug absorption. Or severe GI dysmotility, chronic vomiting, diarrhea, bouts of...
- Clinically significant respiratory disease and/or cardiac disease (medical history or current clinical findings) in the opinion of the investigator.
- Prior treatment cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within...
- QTcF \> 450 msec (men) or QTcF \> 470 msec (women).
See the full eligibility criteria
- Signed Informed Consent
- Males and females aged ≥18 years with a multi-system primary mitochondrial disease.
- 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...
- 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 clinical patient files; AND
- Presence of fatigue (raw total score \>22), assessed by Neuro-QoL SFv1-F at Screening.
- 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...
- 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.
- 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).
- 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...
- Clinically significant respiratory disease and/or cardiac disease (medical history or current clinical findings) in the opinion of the investigator.
- Prior treatment cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to screening.
- QTcF \> 450 msec (men) or QTcF \> 470 msec (women).
- 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 \ 450...
- 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...
- First degree atrioventricular (AV)-block
- Second degree AV-block Type 1 (Mobitz Type 1/Wenckebach type)
- Right bundle branch block.
- History of acute heart failure (within the last 3 months).
- Higher degree of AV-blocks (AVB II° or III°).
- Other exclusion criteria per protocol
The study team makes the final eligibility decision.
Where it's taking place
- Boston, Massachusetts, United States
- Cleveland, Ohio, United States
- Houston, Texas, United States
- Kopenhagen, Region Sjælland, Denmark
- Bordeaux, Gironde, France
- Paris, Paris, France
- München, Germany
- Milan, Italy
- Nijmegen, Gelderland, Netherlands
- London, Greater London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 52 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Boston, Massachusetts, United States; Cleveland, Ohio, United States; Houston, Texas, United States; Kopenhagen, Region Sjælland, Denmark; Bordeaux, Gironde, France; Paris, Paris, France and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.