A safety study of REN001 in mitochondrial myopathy
Research type
Research Study
Full title
An Open-Label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment with Oral REN001 in Patients with Primary Mitochondrial Myopathy (PMM), with an Optional Extension of Treatment
IRAS ID
252961
Contact name
Gráinne Gorman
Contact email
Sponsor organisation
Reneo Pharma Ltd
Eudract number
2018-002969-19
Duration of Study in the UK
1 years, 6 months, 27 days
Research summary
Summary of Research
This is an open label study in approximately 24 subjects with PMM. There will be approximately 12 patients with the m.3243A>G mutation and 12 patients with other mtDNA defects, all subjects must have muscle symptoms (myopathy). The study will evaluate the safety and tolerability of REN001 in subjects with PMM during 12 weeks of treatment (Part A). Subjects may then participate in the optional treatment extension (Part B) if the treatment in Part A was considered safe and well tolerated by the subject and study physician.
Summary of Results
Baseline characteristics:
The age of the patients varied between 43-67 years old, mean 56 years and 70% were female. At baseline, the mean distance walked in a standard Walk Test was 603m and Patient Reported Outcomes (PROs) were abnormal [Modified Fatigue Impact Scale (MFIS) 50.1; Brief Pain Inventory (BPI), 3.5, Short-Form -36 Energy & Fatigue 29.7 compared with normative values of 11, 0 and 60-70, respectively].
Patients enrolled:
Twenty-three (23) patients enrolled into Part A of whom 17 completed, 5 were withdrawn due to Covid-19 pandemic and 1 discontinued for a pre-existing condition. Of the 17 who completed Part A, 13 enrolled into the long-term extension with 11 withdrawn due to Covid-19 after a mean exposure of 27.7 weeks, range 20-40 weeks. Two subjects discontinued for reasons unrelated to study drug and 4 had declined enrolment to Part B for reasons unrelated to dosing.
Safety results:
There were no treatment-related significant side effects. Side effects were typically mild or moderate and no patients stopped their study drug because of side effects. No safety issues were identified for the 13 subjects who continued into Part B and received study drug for up to a total of 40 weeks prior to study termination for pandemic.
Exploratory efficacy results:
Following 12 weeks of 100 mg once-daily dosing with REN001, subjects achieved an average increase of 104 meters in distance walked during the Walk Test compared to baseline. Fifteen of 17 subjects (88%) had an increase in distance walked, with 13 of 17 (76%) increasing by a clinically significant 60 meters or more.
These findings were supported by corresponding improvements in the Patient Reported Outcome Measures of fatigue.REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
18/NE/0309
Date of REC Opinion
29 Oct 2018
REC opinion
Favourable Opinion