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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04788745




Registration number
NCT04788745
Ethics application status
Date submitted
2/03/2021
Date registered
9/03/2021

Titles & IDs
Public title
Targeting Metabolic Flexibility in Amyotrophic Lateral Sclerosis (ALS)
Scientific title
Targeting Metabolic Flexibility in ALS (MetFlex); Safety and Tolerability of Trimetazidine for the Treatment of ALS
Secondary ID [1] 0 0
MetFlex
Universal Trial Number (UTN)
Trial acronym
MetFlex
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 0 0
Motor Neuron Disease 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Trimetazidine Dihydrochloride

Experimental: Experimental - Trimetazidine 35mg


Treatment: Drugs: Trimetazidine Dihydrochloride
Oral tablet, twice-daily

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of Treatment-Emergent Adverse Events; Safety and Tolerability
Timepoint [1] 0 0
16 weeks
Primary outcome [2] 0 0
Level of expression of oxidative stress markers in the plasma and/or serum of trial participants
Timepoint [2] 0 0
16 weeks
Secondary outcome [1] 0 0
Level of expression of oxidative stress markers in the plasma and/or serum of trial participants to inform future clinical trials in ALS/MND
Timepoint [1] 0 0
16 weeks

Eligibility
Key inclusion criteria
* Age between 18 and 75 years
* Signed informed consent prior to the initiation of any study-specific procedures
* Familial or sporadic ALS/MND, defined as clinically possible, probable, or definite as per the El Escorial criteria
* Relative TRICALS risk score between -6.0 to -2.0 (75% of patients with ALS/MND)
* Metabolic index =110%, at the screening visit.
* The use of riluzole will be permitted during the study. Individuals taking riluzole must be on a stable dose for at least 30 days prior to the baseline visit, or stopped taking riluzole at least 30 days prior to the baseline visit.
* Ability to swallow tablets
* Able to lie with torso elevated at a 35° angle for 30 minutes without respiratory support
* Able to give informed consent (as judged by the investigator) and able to comply with all study visits and all study procedures
* Females must not be able to become pregnant (e.g. post-menopausal, surgically sterile or using highly effective birth control methods) for the duration of the study. Highly effective methods of birth control are those with a failure rate of < 1% per year when employed consistently and correctly, e.g. Combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:

* oral
* intravaginal
* transdermal
* Progestogen-only hormonal contraception associated with inhibition of ovulation:
* oral
* injectable
* implantable
* intrauterine device (IUD)
* intrauterine hormone-releasing system ( IUS)
* vasectomised partner
* Females of child-bearing potential must have a negative serum pregnancy test at screening and baseline and be non-lactating
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Unable to provide informed consent
* History of, or current diagnosis of diabetes or medical condition that impacts whole body energy expenditure (e.g. Hashimoto's, heart disease)
* Parkinson's disease or parkinsonism, tremor, restless-leg syndrome
* Safety Laboratory Criteria at screening related to significant kidney disease:

* Creatinine clearance < 50 mL / min (Cockcroft-Gault) based on Cystatin C
* Tracheostomy or non-invasive ventilation (NIV) use > 22 hours per day
* Inability to swallow tablets
* Contraindication therapy:

* Allergy for one of the product's active pharmaceutical ingredients (APIs) or excipients.
* Antihypertensive treatment [Trimetazidine may cause hypotension]
* Evidence of malignant disease
* Significant neuromuscular disease other than ALS/MND
* Ongoing disease that may cause neuropathy
* Pregnancy or breastfeeding
* Females actively seeking to become pregnant who are not using an adequate form of contraceptive as detailed in the Inclusion criteria.
* Deprivation of freedom by administrative or court order

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Royal Brisbane & Women's Hospital - Brisbane
Recruitment postcode(s) [1] 0 0
4029 - Brisbane
Recruitment outside Australia
Country [1] 0 0
Netherlands
State/province [1] 0 0
Utrecht
Country [2] 0 0
United Kingdom
State/province [2] 0 0
London

Funding & Sponsors
Primary sponsor type
Other
Name
The University of Queensland
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
FightMND
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
UMC Utrecht
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
King's College London
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Commercial sector/industry
Name [4] 0 0
Julius Clinical
Address [4] 0 0
Country [4] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Shyuan Ngo, PhD
Address 0 0
The University of Queensland
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.