Open label study of REN001 in PMM subjects
Research type
Research Study
Full title
An open label, multi-centre study to evaluate the long-term safety and tolerability of REN001 in subjects with primary mitochondrial myopathy (PMM)
IRAS ID
304770
Contact name
Lynn Purkins
Contact email
Eudract number
2021-003471-34
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 8 months, 29 days
Research summary
Primary mitochondrial myopathy (PMM) is an illness that results in muscle symptoms including muscle weakness, fatigue and pain. These symptoms are extremely common and often debilitating in patients with PMM. To date, there
are no effective treatments and no cures. Hence, there is an urgent need to find an effective drug treatment. REN001 is a new drug currently under development and has been in five clinical studies. In all these clinical studies, REN001 was considered safe and well tolerated with no safety issues reported. Laboratory and some clinical study data have indicated that REN001 improves energy production within muscle cells and may improve muscle function.This study is being undertaken to evaluate the long-term safety and tolerability of REN001 in subjects with PMM. The study is open to subjects with PMM who have previously completed Study REN001-201 (which is referred to as the STRIDE study) or participated in Study REN001-101 and withdrew from that study due to the COVID-19 pandemic. Eligible subjects will be treated with REN001 100mg orally, once daily for 24 months. The planned maximum duration for each patient in this study will be 24 months treatment and 4 weeks follow up.
REC name
London - Westminster Research Ethics Committee
REC reference
21/LO/0877
Date of REC Opinion
17 Jan 2022
REC opinion
Further Information Favourable Opinion