Actively Recruiting
A Phase III, Randomised, Double-blind, Placebo-controlled Trial to Assess the Efficacy and Safety of Sonlicromanol in Adults with Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation
Led by Khondrion BV · Updated on 2026-01-29
220
Participants Needed
10
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating sonlicromanol in adults with mitochondrial disease caused by a specific genetic mutation (mitochondrial DNA tRNALeu(UUR) m.3243A>G). The study aims to find out if sonlicromanol can reduce fatigue symptoms and their impact on daily life, as well as improve physical abilities like balance and muscle strength. This is a phase III, randomized, double-blind, placebo-controlled trial assessing the efficacy, safety, and tolerability of sonlicromanol over one year. Participants are randomly assigned to receive either 90 mg of sonlicromanol or a matching placebo tablet twice daily for 52 weeks. Neither the participants nor the study team know which treatment is given. The study includes a screening period of up to 4 weeks, followed by the 52-week treatment phase. After finishing treatment, a final follow-up visit is done 2 weeks after the last dose. Up to 220 participants with a confirmed genetic mutation are involved. During the study, participants will complete assessments such as the Neuro-QoL Fatigue Short Form and the Five Times Sit-To-Stand Test at screening, baseline, and several points during treatment. Other evaluations include quality of life surveys and depression inventories. Researchers monitor safety and tolerability throughout. Total participation lasts about 60 weeks, including screening, treatment, and follow-up.
CONDITIONS
Brief Title
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent
- Male or female aged 18 years or older with a multi-system primary mitochondrial disease
- Confirmed mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation with age-adjusted heteroplasmy percentage ≥ 20% in blood, urine, buccal smear, or muscle tissue
- Chronic fatigue lasting at least 3 months not caused by other conditions, with a fatigue score > 22 on Neuro-QoL SFv1-F at screening
- Presence of mitochondrial myopathy: able to complete Five Times Sit-To-Stand Test within 30 seconds and taking ≥ 11 seconds at screening and baseline
- Other inclusion criteria per protocol
You will not qualify if you...
- Treatment with any investigational medical product within 3 months prior to screening or planned use during the study (other than study drug)
- Bone deformities, motor abnormalities, or chronic ulcers that may affect performance in the Five Times Sit-To-Stand Test
- Gastrointestinal surgery or severe GI conditions that may impair drug absorption
- Significant respiratory or cardiac disease or recent interventional cardiac procedures
- QTcF interval > 450 msec (men) or > 470 msec (women)
- Structural heart disease or abnormal heart conduction or repolarization
- History of acute heart failure within 3 months
- Higher degree AV-blocks (second or third degree)
- Family history of unexplained syncope, congenital long or short QT syndrome, or sudden death under age 60
- Other exclusion criteria per protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - Up to 4 weeks
Participants are screened for eligibility to participate in the trial.
Screening and enrollment visit
Duration - 52 weeks
Participants receive either sonlicromanol or placebo tablets twice daily to assess effects on fatigue and physical abilities.
Visits at Baseline (Day 1) and Weeks 13, 26, 39, and 52
Duration - 2 weeks
Participants have a final follow-up visit to assess safety and outcomes after stopping the study medication.
1 visit (in-person)
Trial Site Locations
Total: 10 locations
1
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Not Yet Recruiting
2
Cleveland Clinic Neurological Institute Mellen Center
Cleveland, Ohio, United States, 44195
Not Yet Recruiting
3
The University of Texas Health Science Center at Houston
Houston, Texas, United States, 77030
Not Yet Recruiting
4
Rigshospitalet, University of Copenhagen
Kopenhagen, Region Sjælland, Denmark, DK2100
Not Yet Recruiting
5
CHU de Bordeaux - Hôpital Pellegrin Service Gynecologie Obstetrique
Bordeaux, Gironde, France, 33000
Not Yet Recruiting
6
Groupe Hospitalier Pitie-Salpetriere - Charles-Foix Clinical Investigation Center Paris-Est
Paris, Paris, France, 75013
Not Yet Recruiting
7
Klinikum der Universität München Friedrich-Baur-Institut
München, Germany, 80336
Not Yet Recruiting
8
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Milano, Italy, 20133
Not Yet Recruiting
9
Radboud University Medical Center
Nijmegen, Gelderland, Netherlands, 6525
Actively Recruiting
10
University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery
London, Greater London, United Kingdom, WC1N 3BG
Actively Recruiting
Research Team
J
Jasper Levink, MSc.
G
G. Ruiterkamp, MSc.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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