Status:
RECRUITING
Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS
Lead Sponsor:
Mario Negri Institute for Pharmacological Research
Collaborating Sponsors:
University of Sydney
FightMND
Conditions:
Amyotrophic Lateral Sclerosis
Eligibility:
All Genders
18+ years
Phase:
PHASE2
PHASE3
Brief Summary
Phase II/III multicenter, randomized, double-blind, placebo-controlled trial on acetyl-L-carnitine (ALCAR) in subjects living with amyotrophic lateral sclerosis (ALS). Primary study aim: The clinical ...
Eligibility Criteria
Inclusion
- Age 18+;
- 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 \[Satisfactory bulbar and spinal function (score 3+ on the ALSFRS-R for swallowing, cutting food and handling utensils, and walking)\];
- Satisfactory respiratory function (FVC ≥80% of predicted);
- 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;
- Ability to give written informed consent personally or, as an alternative, via a legally authorized representative;
- Treatment with riluzole 50 mg twice/day for at least 4 weeks prior to randomization visit;
- 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
- 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;
- Other severe clinical conditions (e.g., cardiovascular disorders, neoplasms) with an impact on survival or functional disability in the next 12 months;
- Previous use of ALCAR for any reason;
- Poor compliance with previous treatments;
- 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);
- 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;
- Inability to understand and comply with the study requirements;
- Unwillingness or inability to take riluzole.
Key Trial Info
Start Date :
March 26 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 1 2027
Estimated Enrollment :
246 Patients enrolled
Trial Details
Trial ID
NCT06126315
Start Date
March 26 2025
End Date
September 1 2027
Last Update
September 25 2025
Active Locations (19)
Enter a location and click search to find clinical trials sorted by distance.
1
Concord Hospital
Sydney, Australia, 2139
2
Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII, Dipartimento di Neurologia
Bergamo, Italy, 24127
3
Fondazione Serena ONLUS Centro Clinico NEMO Brescia
Brescia, Italy, 25064
4
Istituto Auxologico Italiano, IRCCS Dipartimento di Neurologia
Milan, Italy, 20149