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Acetyl-L-carnitine to slow functional decline in ALS

A Randomized, Phase II/III Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS

Phase2; Phase3 Interventional Mario Negri Institute for Pharmacological Research · NCT06126315

This trial tests whether two doses of acetyl-L-carnitine can slow loss of independence in adults with recent-onset ALS.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment246 (estimated)
Ages18 Years and up
SexAll
SponsorMario Negri Institute for Pharmacological Research Academic / other
Locations19 sites (Sydney and 18 other locations)
Trial IDNCT06126315 on ClinicalTrials.gov

What this trial studies

This is a multicenter, randomized, double-blind, placebo-controlled Phase II/III trial comparing two doses of acetyl-L-carnitine (1.5 g/day and 3 g/day) against placebo in people with ALS. The primary outcome is progression of functional disability measured by the ALSFRS-R, with secondary outcomes including FVC decline, quality of life (ALSAQ-40), cognition (ECAS), survival without tracheostomy, and biomarker changes. Eligible participants are adults meeting Gold Coast diagnostic criteria with disease duration under 24 months, preserved respiratory function, documented recent progression, and stable riluzole treatment. The trial is conducted at specialist ALS centers in Italy and at Concord Hospital in Sydney, Australia.

Who should consider this trial

Good fit: Adults diagnosed with ALS per Gold Coast criteria within 24 months, with FVC ≥80%, documented functional decline, intact cognition, and on stable riluzole are the ideal candidates.

Not a fit: People with more advanced disease, low respiratory function (FVC <80%), very long disease duration, or significant cognitive impairment are unlikely to meet eligibility or benefit from this trial.

Why it matters

Potential benefit: If successful, the treatment could slow functional decline and help people with ALS maintain independence longer.

How similar studies have performed: Preclinical work and small clinical studies of acetyl-L-carnitine suggest potential neuroprotective effects, but clinical evidence in ALS to date is limited and inconclusive.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Age 18+;
2. ALS diagnosis according to the Gold Coast Criteria;
3. Disease duration \< 24 months from symptom onset, as indicated by limb weakness or bulbar symptoms, at the randomization/baseline visit\*;
4. Self-sufficiency \[Satisfactory bulbar and spinal function (score 3+ on the ALSFRS-R for swallowing, cutting food and handling utensils, and walking)\];
5. Satisfactory respiratory function (FVC ≥80% of predicted);
6. 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.
7. Ability to understand and comply with the study requirements;
8. Ability to give written informed consent personally or, as an alternative, via a legally authorized representative;
9. Treatment with riluzole 50 mg twice/day for at least 4 weeks prior to randomization visit;
10. Intact cognitive function, again determined by the Principal Investigator.

    * The qualifying first symptoms of ALS are limited to manifestations of weakness in extremity, bulbar, or respiratory muscles. Cramps, fasciculations, or fatigue should not be taken in isolation as a first symptom of ALS.

Exclusion Criteria:

1. Antecedent polio infection or other active infection;
2. Motor neuron disease (MND) other than ALS;
3. Involvement of other systems possibly determining a functional impairment (as measured by the endpoints) for the entire duration of the study;
4. Other severe clinical conditions (e.g., cardiovascular disorders, neoplasms) with an impact on survival or functional disability in the next 12 months;
5. Previous use of ALCAR for any reason;
6. Poor compliance with previous treatments;
7. Other experimental treatments in the three months prior to the screening visit (if a subject is receiving another experimental drug, a 3-month wash-out period before participating in the present clinical trial will be required);
8. Women who are lactating or able to become pregnant (e.g. who are not post-menopausal, surgically sterile, or using inadequate birth control) and men unable to practice contraception for the duration of the treatment and three months after its completion;
9. Inability to understand and comply with the study requirements;
10. Unwillingness or inability to take riluzole.

Where this trial is running

Sydney and 18 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
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Conditions Amyotrophic Lateral Sclerosis
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.