Phase 2b Safety and Efficacy Study of REN001 in Mitochondrial Myopathy
Research type
Research Study
Full title
A double-blind, placebo-controlled, study to evaluate the efficacy and safety of 24 weeks treatment with REN001 in patients with Primary Mitochondrial Myopathy (PMM)
IRAS ID
286650
Contact name
Lynn Purkins
Contact email
Sponsor organisation
Reneo Pharma Ltd
Eudract number
2020-002855-40
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 6 months, 0 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, to date 106 people have been given REN001 in four 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.
The planned maximum duration for each patient in this study will be 36 weeks (8 weeks screening, 24 weeks treatment and 4 weeks follow up). Each patient will only be given either REN001 treatment or placebo (dummy medication) treatment for 24 weeks. Neither the doctor nor the patient will know who is given which study drug treatment (double-blind). This is important to ensure that any effects are due to the medication and not to any other potential factors (e.g. more interaction with medical staff or hospital visits).
This study will determine how well REN001 works and whether it is safe and well tolerated in patients with PMM.REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
21/NE/0077
Date of REC Opinion
12 May 2021
REC opinion
Further Information Favourable Opinion