Randomised double-blinded control adaptive trial to measure of the effectiveness of acipimox in patients with Mitochondrial Myopathy

Phase 1

• Conditions: Mitochondrial Myopathy; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2018-002721-29-GB
• Lead Sponsor: ewcastle upon Tyne Hospitals NHS Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-06-18
• Last Update Posted: 16 December 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 120

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:
i) Genetically proven diagnosis of m.3243A>G mutation or single large-scale mtDNA deletion, and
ii)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.

Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 70
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 40

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:
a)pregnant
b)breast feeding
c)of childbearing potential with a positive urine pregnancy test
prior to starting trial IMP or;
d) 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 clinical trial of medicinal product
• Study Design: Not specified