Safety and tolerability of Trimetazidine in amyotrophic lateral sclerosis (ALS)
- Conditions
- Patients suffering from amyotrophic lateral sclerosis (ALS)Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2020-005018-17-NL
- Lead Sponsor
- The University of Queensland
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 36
- 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, probable laboratory supported 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 (defined as measured resting energy expenditure as a % of predicted resting energy expenditure) =110%, averaged over the lead-in period.
- 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 highley effective birth control methods) for the duration of the study.
- Females of child-bearing potential must have a negative serum pregnancy test at screening and baseline and be non-lactating
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 16
- 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 (Cockroft-Gault) based on Cystatin C
- Tracheostomy or non-invasive ventilation (NIV) use > 22 hours per day
- Contraindication therapy:
Allergy for one of the product's API's or expedients.
Antihypertensive treatment [Trimetazidine may cause hypotension]
- Evidence of malignant disease
- Significant neuromuscular disease other than ALS/MND
- Ongoing disease that may cause neuropathy
- 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
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method