NCT04788745

Targeting Metabolic Flexibility in Amyotrophic Lateral Sclerosis (ALS)

Targeting Metabolic Flexibility in ALS (MetFlex); Safety and Tolerability of Trimetazidine for the Treatment of ALS

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: The University of Queensland · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

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 (ALS/MND).

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 trimetazidine for 12 weeks. Participants will visit the clinic at 6-week intervals, during which we will obtain a blood sample to measure the pharmacodynamic response. We will also collect information regarding the rate of disease progression (i.e. ALSFRS-R and SVC). At weeks 3 and 9 of treatment, participants will conduct a teleconference visit, during which we will collect data on ALSFRS-R. Adverse events will be collected and recorded throughout the entire trial duration. At the end of the on-treatment period, a close-out visit will occur after four weeks. The total study period per participant will be 20 weeks.

Conditions

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease

Interventions

Type	Name	Description
DRUG	Trimetazidine Dihydrochloride	Oral tablet, twice-daily

Timeline

• Start date: 2021-06-29
• Primary completion: 2023-05-24
• Completion: 2023-05-24
• First posted: 2021-03-09
• Last updated: 2023-07-20

Locations

3 sites across 3 countries: Australia, Netherlands, United Kingdom