What is this trial about?
REN001 is a new drug currently under development, it has previously been given to a limited number of healthy volunteers and patients with high lipids levels in controlled clinical trials. Laboratory and some clinical trial data have indicated that REN001 improves energy production by mitochondria within muscle cells and may improve muscle function.
The aim of this trial is to see whether treatment with REN001 is safe and well tolerated in primary mitochondrial myopathy (PMM) patients. As part of the trial there will also be some tests to see what is the best way to measure muscle symptoms in PMM patients.
Who can take part?
UK Patients aged 16 years and over, men or women, with a clinical and genetic diagnosis of:
Mitochondrial disease caused by either the 3243A>G mutation in mitochondrial DNA or other mitochondrial DNA defect (where a piece of mitochondrial DNA is damaged or changed) and must also have evidence of muscle symptoms.
Participants must not be involved in any other ongoing clinical trial research.
There are certain circumstances where some people might not be able to participate in this trial, so the research team will carry out a thorough screening check to make sure patients are eligible.
What's involved in taking part?
The trial will consist of two parts with a number of trial visits in each part, some visits may be done at home by a qualified nurse if you prefer to avoid the inconvenience of travelling to the trial centre. The two parts of the trial and the visit schedule are given in more detail below.
Part A: Participants will be given 12 weeks (3 months) treatment with REN001 with the visit schedule listed below:
- Screening visit (1 day at trial centre)
- Baseline visit (3 days at trial centre)
- Week 1 (≈ 2 hours can be done at home)
- Week 2 (≈ 2 hours at trial centre)
- Week 4 and Week 8 (≈ 2 hours can be done at home)
- Week 12 visit (3 days at trial centre)
- Follow Up (≈ 2 hours can be done at home) – Only if not entering Part B of the Trial
Most visits will only include blood tests and brief questionnaires. The baseline visit and the visit at the end of 12 weeks will be more intensive and will take place over (up to) 3 days. These tests include two small skeletal muscle biopsies (one at the start of the study and one at the end), blood tests, walking and stationary bike cycling exercise tests and several questionnaires. These tests will be done to determine whether REN001 is safe and whether there are any muscle changes
Following 12 weeks of treatment, and with agreement from your trial doctor, you may be able to continue taking REN001 for an additional 36 weeks (9 months), if both you and your doctor believe that taking REN001 has been beneficial to you.
Part B: Participants will be given a further 36 weeks (9 months) treatment with REN001 with the visit schedule listed below:
- Week 16 and Week 20 (≈ 2 hours can be done at home)
- Week 24 (≈ 4 hours at trial centre)
- Week 28 and Week 32 (telephone call at home)
- Week 36 visit (≈ 4 hours at trial centre)
- Week 40 and Week 44 (telephone call at home)
- Week 48 (3 days at trial centre)
- Follow Up (≈ 2 hours can be done at home)
Again, most visits will only include blood tests and brief questionnaires. Further tests will be done including blood tests, functional/exercise tests and several questionnaires at the Week 48 visit. No further muscle biopsies will be taken in Part B.
Will there be any costs associated with my participation in the trial?
Patients participating in the trial will be supported with the option of home nursing visits for some trial visits if they would prefer not to travel to the trial centre. Also, to ease the burden of travelling to and from visits, there will be a specialist company who will book and organise payment for travel and hotels for you and who will also ensure you are promptly reimbursed for any other trial expenses e.g. refreshments. In addition, as there may be no health benefit for participating in the trial, patients will also be paid for Part A to compensate them for their time in participating in trial visits.
Where is the trial taking place?
The trial is funded by Reneo Pharma Ltd and is being run at both the Newcastle upon Tyne Hospital and at University College London (UCL). Participants do not have to be patients at these hospitals to take part in this trial. Participants can be seen at either the Newcastle or UCL hospitals.
Are there any risks?
The trial involves a series of standard clinical tests that are routinely carried out in clinical practice. This includes muscle biopsies, blood samples, and functional and exercise tests. Muscle biopsies are often used to diagnose mitochondrial disease and you may well have had one before. We will give you a local anaesthetic injection which can cause some stinging pain, and if you feel any pain during the rest of the procedures we will give additional anaesthetic or abandon the procedure if you’d prefer us to stop. There is a risk of bruising and bleeding and you will feel sore around the site for a few days. Taking blood samples may cause some discomfort or bruising and can cause some people to faint.
This is the first trial where REN001 has been given to patients with PMM, so there is no information available about any potential side effects in PMM patients. However, REN001 has been given to healthy volunteers in 2 previous clinical trials and to obese patients in another clinical trial. REN001 was considered safe and well tolerated in these previous trials. The most common side effects in these studies were:
- Headache (occurred in greater than 1 in 100 but less than 1 in 10 subjects)
- Rash (occurred in greater than 1 in 100 but less than 1 in 10 subjects)
However as with any treatment, side effects that have not happened before may occur.
Who will benefit?
There is no direct benefit to you from taking part in this research. However, knowledge gained from the trial may ultimately improve the treatment of mitochondrial disease.
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 trial team directly:
Newcastle Team (Ann McNichol) on:
Tel: 0191 2820070
Email: [email protected]
UCL Team (Iwona Skorupinska or Louise Germain) on :
Tel: 0203 108 7527
Email: [email protected]
Email: [email protected]
