Acetyl-L-carnitine to slow functional decline in ALS

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Concord Hospital
  Sydney, Australia, Australia Recruiting
• Fondazione Serena ONLUS Centro Clinico NEMO Brescia
  Brescia, Brescia, Italy Recruiting
• Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII, Dipartimento di Neurologia
  Bergamo, Italy, Italy Recruiting
• Istituto Auxologico Italiano, IRCCS Dipartimento di Neurologia
  Milan, Italy, Italy Recruiting
• Azienda Ospedaliera Universitaria Federico II Di Napoli
  Naples, Italy, Italy Recruiting
• Azienda Ospedaliero-Universitaria Maggiore della Carità
  Novara, Italy, Italy Recruiting

Study contacts

• Study coordinator: Elisabetta Pupillo, PharmD
• Email: elisabetta.pupillo@marionegri.it
• Phone: 00390239014605

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.