Can the drug acipimox relieve muscle symptoms in patients with mitochondrial myopathy?

Not Applicable

Completed

• Conditions: Mitochondrial myopathy; Nervous System Diseases

• Registration Number: ISRCTN12895613
• Lead Sponsor: The Newcastle Hospitals NHS Foundation Trust

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-01-03
• Study Completion: 2022-01-19
• Last Update Posted: 10 October 2022

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 21

Inclusion Criteria

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.

Exclusion Criteria

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
7. Patients with a platelet count of < 50 platelets/ ul of blood
8. Patients on treatment with methotrexate or other immunosuppressant medications
9. Patients with active known peptic ulcer or history of recurrent ulceration
10. Patients on treatment with warfarin, clopidogrel, regular high-dose (> = 300 mg OD) aspirin or other anticoagulant medications which in the opinion of the investigator precludes entry into the trial. Patients receiving high-dose aspirin who are able to come off aspirin for a period of 72 hours prior to any muscle biopsy sample will be eligible to participate
11. Patients with a medical history which in the opinion of the investigator contraindicates the use of low-dose aspirin
12. Patients who are already taking acipimox
13. Patients with an elective hospital admission scheduled during the trial period, which in the opinion of the investigator would preclude participation

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
ATP content in skeletal muscle will be measured from biopsy specimens using a luminescence assay at baseline and 12 weeks.