Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS

What Was Tested

This registered phase 2, phase 3 trial tested L-Carnitine.

Phase II/III multicenter, randomized, double-blind, placebo-controlled trial on acetyl-L-carnitine (ALCAR) in subjects living with amyotrophic lateral sclerosis (ALS).

The effect of ALCAR treatment on different clinical aspects: functional decline as measured by ALSFRS-R total score; the decline of forced vital capacity (FVC); quality of life as measured by ALSAQ-40 scale; cognitive function as measured by Edinburgh Cognitive and Behavioural ALS Screen (ECAS) scale; survival (being alive and without tracheostomy). 2.

It had three arms (groups): one (experimental), one (experimental) and placebo (placebo comparator).

2 pockets of placebo will be administered t.i.d for 48 weeks.

The registry classes the interventions as drug.

Who Took Part

246 planned participants were the target.

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: ALS diagnosis according to the Gold Coast Criteria;. Disease duration \< 24 months from symptom onset, as indicated by limb weakness or bulbar symptoms, at the randomization/baseline visit\*;. Self-sufficiency \[Satisfactory bulbar and spinal function (score 3+ on the ALSFRS-R for swallowing, cutting food and handling utensils, and walking)\];. Documented progression of symptoms as measured by the ALSFRS-R scale. Disease progression rate (DFS) must be\>= 0.33. DFS =(48- ALSFRS-R at screening)/months from onset to screening. Ability to understand and comply with the study requirements;.

The full record lists 11 inclusion and 10 exclusion criteria; among the exclusions: antecedent polio infection or other active infection;; motor neuron disease (mnd) other than als;; involvement of other systems possibly determining a functional impairment (as measured by the endpoints) for the entire duration of the study;.

How the Study Was Run

It was run as a randomized, parallel-group and quadruple-blind study.

Because people in this L-Carnitine study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

The L-Carnitine group and the comparison group ran side by side rather than switching.

Blinding on both sides kept the participants and researchers unaware of who received L-Carnitine.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2025-03-26 with completion listed for 2027-04.

It ran at 19 sites in Italy (18) and Australia (1).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: self-sufficient (time frame: 48 weeks).

It also tracked 11 secondary outcomes, for example Mean change of ALSFRS-R total score in each treatment arm, Mean change of FVC% in each treatment arm, Mean change in ECAS total score in each treatment arm, Cumulative probability of remaining self-sufficient in each treatment arm, Cumulative probability of remaining free from a 6-point or greater decline in ALSFRS-R total score in each treatment arm and Cumulative probability of remaining without gastrostomy in each treatment arm.

What This Means in Plain Language

Put simply, this is a phase 2, phase 3, randomized trial of L-Carnitine in 246 people.

It is still recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the L-Carnitine compound page, all human trials.