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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
29/06/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
24/05/2023
Sample size
Target
Accrual to date
Final
21
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.


Results not provided in https://clinicaltrials.gov/study/NCT04788745