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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06126315




Registration number
NCT06126315
Ethics application status
Date submitted
6/11/2023
Date registered
13/11/2023
Date last updated
25/06/2025

Titles & IDs
Public title
Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS
Scientific title
A Randomized, Phase II/III Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS
Secondary ID [1] 0 0
ALCALS
Universal Trial Number (UTN)
Trial acronym
ALCALS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Acetyl-l-carnitine
Treatment: Drugs - Placebo

Experimental: alcar 1.5 g - 2 pockets of ALCAR will be administered t.i.d for 48 weeks. Total daily dosage: 1.5 g

Experimental: alcar 3 g - 2 pockets of ALCAR will be administered t.i.d for 48 weeks. Total daily dosage: 3 g

Placebo comparator: placebo - 2 pockets of placebo will be administered t.i.d for 48 weeks.


Treatment: Drugs: Acetyl-l-carnitine
Acetyl-l-carnitine

Treatment: Drugs: Placebo
placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
self-sufficient
Timepoint [1] 0 0
48 weeks
Secondary outcome [1] 0 0
Mean change of ALSFRS-R total score in each treatment arm
Timepoint [1] 0 0
from baseline to week 48
Secondary outcome [2] 0 0
Mean change of FVC% in each treatment arm
Timepoint [2] 0 0
from baseline to week 48
Secondary outcome [3] 0 0
Mean change in the five domains of ALSAQ-40 measuring different aspects of quality of life (physical mobility, ADL/independence, eating and drinking, emotional reactions, communication) in each treatment arm
Timepoint [3] 0 0
from baseline to week 48
Secondary outcome [4] 0 0
Mean change in ECAS total score in each treatment arm
Timepoint [4] 0 0
from baseline to week 48
Secondary outcome [5] 0 0
Cumulative probability of remaining self-sufficient in each treatment arm
Timepoint [5] 0 0
from baseline to week 48
Secondary outcome [6] 0 0
Cumulative probability of remaining free from a 6-point or greater decline in ALSFRS-R total score in each treatment arm
Timepoint [6] 0 0
from baseline to week 48
Secondary outcome [7] 0 0
Cumulative probability of remaining without gastrostomy in each treatment arm
Timepoint [7] 0 0
from baseline to week 48
Secondary outcome [8] 0 0
Cumulative probability of remaining without non-invasive ventilation (NIV) support (=12 hours a day in a 24-hour period) in each treatment arm
Timepoint [8] 0 0
from baseline to week 48
Secondary outcome [9] 0 0
Cumulative survival probability (of being alive and without tracheostomy) in each treatment arm
Timepoint [9] 0 0
from baseline to week 48
Secondary outcome [10] 0 0
The mean change in the levels of PGC-1 alpha, 3-NT, acetyl-PPIA in the peripheral blood mononuclear cells (PBMCs) and of NFL, MMP-9, MCP-1, CK, MiR-206, Musclin/osteocrin, Uric acid, HNE in plasma in each treatment arm, during the entire treatment period
Timepoint [10] 0 0
from baseline to week 48
Secondary outcome [11] 0 0
Number of adverse events and serious adverse events in each treatment arm
Timepoint [11] 0 0
from baseline to week 48

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/03/2025
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2027
Actual
Sample size
Target
246
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Concord Hospital - Sydney
Recruitment postcode(s) [1] 0 0
2139 - Sydney
Recruitment outside Australia
Country [1] 0 0
Italy
State/province [1] 0 0
Bergamo
Country [2] 0 0
Italy
State/province [2] 0 0
Brescia
Country [3] 0 0
Italy
State/province [3] 0 0
Milano
Country [4] 0 0
Italy
State/province [4] 0 0
Modena
Country [5] 0 0
Italy
State/province [5] 0 0
Napoli
Country [6] 0 0
Italy
State/province [6] 0 0
Novara
Country [7] 0 0
Italy
State/province [7] 0 0
Padova
Country [8] 0 0
Italy
State/province [8] 0 0
Palermo
Country [9] 0 0
Italy
State/province [9] 0 0
Pavia
Country [10] 0 0
Italy
State/province [10] 0 0
Pergine Valsugana
Country [11] 0 0
Italy
State/province [11] 0 0
Perugia
Country [12] 0 0
Italy
State/province [12] 0 0
Pisa
Country [13] 0 0
Italy
State/province [13] 0 0
Roma
Country [14] 0 0
Italy
State/province [14] 0 0
Torrette

Funding & Sponsors
Primary sponsor type
Other
Name
Mario Negri Institute for Pharmacological Research
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
University of Sydney
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
FightMND
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Elisabetta Pupillo, PharmD
Address 0 0
Istituto Di Ricerche Farmacologiche Mario Negri
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Elisabetta Pupillo, PharmD
Address 0 0
Country 0 0
Phone 0 0
00390239014605
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT06126315