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 - A safety study of REN001 in mitochondrial myopathy

Reneo Pharma Ltd.0 sites24 target enrollmentSeptember 12, 2018

Overview

No summary available.

Registry

who.int

Start Date

September 12, 2018

End Date

TBD

Last Updated

6 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

•Subjects must meet all of the following inclusion criteria to be eligible for inclusion in the study:
•1\. Males or females aged 16 years or older.
•2\. Established diagnosis of PMM according to the 2016 Rome Consensus recommendations (Mancuso, McFarland, Klopstock, \& Hirano, 2017\).
•3\. A score of 2\-4 on Question 5 Section III of NMDAS from subject's medical history or at screening whichever is most relevant.
•4\. Either a confirmed m.3243A\>G mutation or other mtDNA defects, with myopathy.
•5\. Have no changes to their exercise regimen within 30 days prior to Day 1 and be willing to remain on the same exercise regimen for the duration of the study.
•6\. Subjects must be ambulatory and able to perform the study exercise tests independently.
•7\.Subjects must be able to remain on stable medication through the study and specifically must not commence or have changes in medication for diabetes.
•8\. Have adequate kidney function defined as an estimated glomerular filtration rate (eGFR) \= 60 mL/min/1\.73 m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD\-EPI) creatinine equation (Levey, et al., 2009\) at Screening and Baseline.
•9\. Be able to swallow capsules.

•Subjects presenting with any of the following will not be included in the study:
•1\. Participation in a prior REN001 (previously known as HPP\-593\) study.
•2\. Employee of the study site or of the Sponsor’s company or delegates.
•3\. Documented evidence of ongoing rhabdomyolysis.
•4\. Currently taking or anticipated to need a PPAR agonist during the study.
•5\. Subjects with motor abnormalities other than related to the mitochondrial disease that may interfere with the outcome measures.
•6\. Treatment with an investigational drug within 3 months prior to Day 1\.
•7\. Currently taking or anticipated to need a prescription and/or non\-prescription drug that might interfere with the study endpoints.
•8\. Evidence of significant concomitant clinical disease that may need a change in management during the study or could interfere with the conduct or safety of this study (Stable well\-controlled chronic conditions such hypercholesterolemia, gastroesophageal reflux, or depression under control with medication (other than tricyclic antidepressants), are acceptable provided the symptoms and medications would not be predicted to compromise safety or interfere with the tests and interpretations of this study).
•9\. Subjects with diabetes who require treatment with insulin who are unwilling to undertake glucose monitoring for at least the first 12 weeks of dosing or in the Investigators opinion will not be able to adequately maintain glucose homeostasis.