What is this study about?
The ‘STRIDE AHEAD’ study is an open-label extension trial for patients who have already taken part in the STRIDE study or in Study REN001-101. By joining the new study, patients who have participated in the earlier REN001 studies in mitochondrial myopathy will now have the chance to receive the active drug. The STRIDE AHEAD study aims to evaluate the safety and tolerability of REN001 when given to patients over a longer period of time (24 months).
Who can take part?
Patients with Primary Mitochondrial Myopathy (PMM) who have already taken part in the REN001-201 (STRIDE) study, or who took part in the REN001-101 study and had to withdraw from the study due to the COVID-19 pandemic. As with the earlier studies, eligible patients will need to be aged 18 years or over and have a genetic diagnosis of PMM. They must not be taking part in any other clinical trial. Potentially eligible patients will need to go through certain screening checks to make sure that they still meet the list of requirements needed to take part in this study.
What drug is being tested?
The active drug being tested is called REN001. In STRIDE AHEAD, everyone will receive the active drug. This involves taking 2 gelatin capsules of REN001 once daily with food for 24 months.
What’s involved in taking part?
There are several enrolment options for STRIDE patients, you can discuss with your physician which enrolment option would work best for you:
-
Directly from STRIDE after completing week 24
-
At the STRIDE follow up visit
-
After the STRIDE follow up visit
For patients who took part in REN001-101, you can contact the study site and they will be able to discuss enrolment with you.
The different enrolment options will require different health checks to make sure it is appropriate for you to enter the STRIDE AHEAD study, and this may require you to visit the site for 1 or 2 occasions. The study site will be able to explain if these visits are needed for you to enrol.
Once you have been enrolled in the study there will be 6 visits at the study centre over 24 months:
- 1 Month Visit: Approximately 1.5 hours visit
- 3 Month Visit: Approximately 8 hours at the study centre
- 6, 12, 18 and 24 Month Visits will each be approximately 8 hours at the study centre.
Most visits will include blood tests, questionnaires and a walk test. These tests will be done to evaluate the long-term safety and effectiveness of REN001.
Will there be any costs associated with my participation in the study?
To ease the burden of travelling to and from visits, the study site staff or a specialist company will book and organise payment for travel and hotels for you and will also ensure that you are promptly reimbursed for any other study-related travel expenses (e.g. refreshments or meals).
Where is the study taking place and who is sponsoring this study?
As with the REN001-101 and REN001-201 (STRIDE) studies, STRIDE AHEAD is being funded by Reneo Pharma Ltd and is taking place in 2 centres in the UK, Newcastle and University College London (UCL).
Are there any risks?
The study involves a series of clinical tests that are routinely carried out in clinical practice. This includes blood samples and exercise tests. Taking blood samples may cause some discomfort or bruising and can cause some people to faint. All of the study and exercise tests are routinely used in the clinics.
REN001 has been given to healthy volunteers, to obese patients and to patients with PMM in previous clinical studies. REN001 was considered to be safe and well tolerated in all these trials. In the PMM patient study, the most common side effects reported were:
- Constipation
- Headache
However, as with any treatment, side effects that have not been previously observed may occur.
Who will benefit?
We hope that the information that we collect from this study will help us understand whether this drug may be useful in treating the muscle symptoms of mitochondrial disease.
How do I find out more?
Newcastle :
Clinical Research Nurse, Clinical Ageing Research University, Newcastle University, Campus for Ageing and Vitality, NE4 5PL
Contact: Ann McNichol
Tel: 0191 2081250
UCL:
Queen Square Centre for Neuromuscular Diseases, 8-11 Queen Square, London, WC1N 3BG
Contact: Iwona Skorupinska
Tel: :02031087515
Or
Louise Germain
Phone: 0203 108 6308
