Randomised, double-blinded, placebo-controlled, adaptive design trial of the efficacy of acipimox in patients with Mitochondrial Myopathy

The Newcastle Hospitals NHS Foundation Trust0 sites21 target enrollmentJanuary 3, 2019

Overview

2022 Protocol article in https://pubmed.ncbi.nlm.nih.gov/36127727/ (added 27/09/2022)

Registry

who.int

Start Date

January 3, 2019

End Date

January 19, 2022

Last Updated

3 years ago

Study Type

Interventional

Sex

All

Sponsor

The Newcastle Hospitals NHS Foundation Trust

•1\. Must be able to provide full informed consent
•2\. Male or female patients \= 16 years of age
•3\. Patients must fulfil the following:
•3\.1\. Genetically proven diagnosis of m.3243A\>G mutation or single large\-scale mtDNA deletion, and
•3\.2\. Evidence of myopathy as confirmed by the investigator
•4\. Able and willing, in the opinion of the investigator, to comply with all trial requirements
•5\. Willing for their GP and Specialist (if applicable), to be informed of their participation in the trial
•6\. Be on a stable dose of any current regular medication for at least four weeks prior to trial entry.

•1\. Patients who are currently participating or have participated in a clinical trial of an investigational medicinal product within the 12\-week period prior to the date of informed consent
•2\. Patients who have had an elective or emergency admission to hospital within the 4\-week period prior to the date of informed consent
•3\. Patients with other known uncontrolled medical problems, which, in the opinion of the investigator, would preclude participation in the trial
•4\. Patients who are:
•4\.1\. Pregnant
•4\.2\. Breastfeeding
•4\.3\. Of childbearing potential with a positive urine pregnancy test prior to starting trial IMP
•4\.4\. Male or female of childbearing potential unwilling to use a double barrier method of contraception throughout the trial (postmenopausal women must be amenorrhoeic for at least 12 months to be considered of non\-childbearing potential)
•5\. Patients with moderate to severe renal impairment (creatinine clearance \< 60 ml/min)
•6\. Patients with a screening AST, ALT or Gamma GT result of more than 3 times the upper limit of normal