What is this study about?

KHENERFIN is a Phase 3 study investigating whether sonlicromanol can help people with Primary Mitochondrial Disease (PMD) caused by the m.3243A>G variant in the MT‑TL1 gene including MELAS, MIDD and other multisystem forms of the disease. The trial is investigating whether the treatment can reduce physical fatigue, improve leg muscle strength and balance and enhance overall quality of life for people living with PMD.

Who can take part?

You may be eligible to participate if you:

• Are ≥18 years of age
• Have genetically confirmed m.3243A>G-related multi-system Primary Mitochondrial Disease
• Have reported chronic fatigue (not attributable to other aetiologies) for ≥3 months prior to screening

Final eligibility will be determined by the study doctor after the screening visit.

What drug is being tested?

Sonlicromanol is the investigational medicine that is tested in this study. Sonlicromanol has been studied in five clinical trials and has been used over 3 years in compassionate use programs. Sonlicromanol will be provided as dispersible oral tablets.

All participants will be equally divided in two groups. Eligible participants will be randomly assigned (like picking straws) to receive either the study medicine (sonlicromanol) or placebo (no active medication). Neither participants nor researchers know who is receiving which treatment during the study.

What’s involved in taking part?

You will receive a placebo or investigational drug for 52 weeks. During the study you will visit the clinic nine times.

During the clinic visits you will be asked to complete questionnaires about your (mental) health, pain, your energy levels and fatigue and you will undergo a series of tests and procedures, such as an ECG and echocardiogram, blood sampling, a test of strength and endurance of your lower body (5 x sit-to-stand test).

During the study visits you will also discuss any side-effects you may be experiencing and any medications and supplements you have been taking.

Will there be any costs associated with my participation in the study?

There are no costs for you if you take part. You will receive the study drug or placebo at no cost, and you will not be charged for any study-related procedures.

Reasonable travel and subsistence costs, such as fuel, bus or train tickets, will be reimbursed with valid receipts. If you agree, a specialist travel company will provide personalised travel support. The company can arrange your transport and hotel bookings and reimburse related expenses, including meals, parking and mileage.

Where is the study taking place?

In the UK, the following site has started recruiting:

• UCL Queen Square Institute of Neurology – PI: Prof. Robert Pitceathly, Professor of Clinical Neurology and Mitochondrial Medicine. 

Are there any risks?

The study involves a series of clinical tests that are often carried out in clinical practice. This includes blood samples, cognitive and exercise tests. Taking blood samples may cause some discomfort or bruising and can cause some people to faint.

Sonlicromanol has been given to healthy volunteers and to primary mitochondrial disease patients in previous clinical studies. In all these clinical studies, sonlicromanol was considered safe and well tolerated with no safety issues reported.

Everyone taking part in the study will be monitored for any adverse effects, however the study team does not know all the effects that the study drug may possibly have on you and side-effects that have not happened before may occur.

Who will benefit?

You may or may not benefit from taking part in this study. However, in the future other people may benefit from this research.

How do I find out more?

If you think you might be eligible and are interested in taking part in this research, or would like any further information, please contact the study team directly:

London:

UCL Queen Square Institute of Neurology

Louise Germaine: [email protected].