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

Launched by MARIO NEGRI INSTITUTE FOR PHARMACOLOGICAL RESEARCH · Nov 6, 2023

Keywords

ClinConnect Summary

This clinical trial is studying the effects of a treatment called acetyl-L-carnitine (ALCAR) on people with amyotrophic lateral sclerosis (ALS), a disease that affects the nerves and can lead to loss of muscle control. The trial will test two different doses of ALCAR (1.5 grams and 3 grams per day) to see if it can slow down the progression of the disease, which is measured by how well patients can perform daily activities. Along with this, the trial will look at how ALCAR affects other aspects of health, like breathing ability, quality of life, and cognitive function.

To participate, individuals must be at least 18 years old, have a confirmed diagnosis of ALS for less than 24 months, and show some ability to perform daily tasks like swallowing and walking. They also need to have good respiratory function. Participants will be randomly assigned to receive either ALCAR or a placebo (a treatment that doesn't contain active medication) for 48 weeks, in addition to another medicine called riluzole. Throughout the study, they will attend several follow-up visits where their health will be monitored, and researchers will collect blood samples to measure certain biological markers. This trial aims to understand both the effectiveness and safety of ALCAR for ALS patients.

Gender

ALL

Eligibility 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.