Status:
COMPLETED
Targeting Metabolic Flexibility in Amyotrophic Lateral Sclerosis (ALS)
Lead Sponsor:
The University of Queensland
Collaborating Sponsors:
FightMND
UMC Utrecht
Conditions:
Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Eligibility:
All Genders
18-75 years
Phase:
PHASE2
Brief Summary
MetFlex is an investigator led, open-label, single-arm, Phase 2a trial to determine the safety and tolerability of trimetazidine for the treatment of amyotrophic lateral sclerosis/motor neuron disease...
Detailed Description
The study will consist of a 4-week lead-in period to obtain a stable baseline measurement of clinical markers of disease and oxidative stress. After the lead-in phase, participants will receive trimet...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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
Key Trial Info
Start Date :
June 29 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 24 2023
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT04788745
Start Date
June 29 2021
End Date
May 24 2023
Last Update
July 20 2023
Active Locations (3)
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1
Royal Brisbane & Women's Hospital
Brisbane, Queensland, Australia, 4029
2
University Medical Centre Utrecht
Utrecht, Netherlands
3
King's College London
London, United Kingdom