The goal of this clinical trial is to evaluate whether low-dose colchicine can slow disease progression in patients with amyotrophic lateral sclerosis (ALS), a progressive and fatal neurodegenerative disorder affecting motor neurons.
The study is designed to answer whether patients receiving colchicine show a slower decline in functional status, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), over a 30-week double-blind treatment period compared to patients receiving placebo. Additional questions include whether colchicine has an effect on respiratory function, disability progression, quality of life, and overall survival.
Researchers will compare participants receiving colchicine at a dose of 0.005 mg/kg/day with those receiving placebo, both in addition to standard-of-care therapy with riluzole, to assess potential differences in disease progression.
Participants will be randomly assigned in a 2:1 ratio to colchicine or placebo. They will take the assigned study medication for 30 weeks during a double-blind phase and then continue into a 36-week open-label extension phase, during which all participants will receive colchicine while remaining blinded to their initial treatment assignment. Throughout the study, participants will undergo regular clinical evaluations, including assessments of motor and respiratory function, functional disability, and quality of life, for a total follow-up period of up to 66 weeks. Blood samples will also be collected to investigate biological markers of neurodegeneration and inflammation.
Patient age strictly between 18 and 80 years at the time of screening.
Definitively established diagnosis of ALS (sporadic or familial) matching standardized clinical consensus parameters.
Stable background regimen of European gold-standard Riluzole therapy maintained at a fixed dose of 100 mg/day for a minimum of 1 month prior to baseline randomization.
BMI>17.5 Kg/m2
Concurrent participation or treatment within any other interventional or drug-based clinical trial.
Clinically significant hepatic impairment (defined as baseline serum transaminases AST or ALT exceeding 3x Upper Limit of Normal [ULN], or total bilirubin exceeding 2x ULN).
Severe renal insufficiency, documented bone marrow suppression, or significant hematological abnormalities.
Known hypersensitivity or systemic intolerance to colchicine or any of the manufacturing excipients (lactose, sucrose, magnesium stearate, arabic gum).
Parallel
Colchicine 0.5 MG Oral Tablet
DrugLow-dose colchicine administered orally at 0.005 mg/kg/day as an add-on to standard-of-care therapy with riluzole. Participants receive colchicine once daily or every other day depending on body weight (≥70 kg: 0.5 mg daily; \<70 kg: 0.5 mg every other day) using matching oral tablets. The intervention is administered during a 30-week double-blind phase followed by a 36-week open-label extension phase.
Placebo Oral Tablet
DrugMatching placebo oral tablets identical in appearance, taste, and administration schedule to colchicine. Participants receive placebo in addition to standard-of-care therapy with riluzole. The placebo is administered orally once daily or every other day depending on body weight, following the same dosing schedule as the active treatment arm, during the 30-week double-blind phase. Participants subsequently enter a 36-week open-label extension phase during which all participants receive active colchicine.
Changes in ALS disease progression as measured by ALS Functional rating Scale Revised (ALSFRS-R)
To assess whether low-dose colchicine slows disease progression in ALS by comparing the monthly rate of decline in the ALS Functional Rating Scale-Revised (ALSFRS-R) total score between the colchicine and placebo groups during the double-blind treatment phase.
Longitudinal change in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score
Change in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score from baseline over time in both treatment groups. The ALSFRS-R total score ranges from 0 to 48, with higher scores indicating better functional status.
Change in ROADS score
Change in Changes in functional disability, assessed using the Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS) score from baseline over time in both treatment groups. The ROADS score ranges from 0 to 100, with higher scores indicating better functional status.
Tracheostomy-free survival rate
Overall survival from randomization to date of death or tracheostomy
Change in Forced Vital Capacity (FVC)
Changes in FVC score from baseline over time in both treatment groups.
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Azienda Ospedaliero-Universitaria di Modena
Lead sponsor
University of Milan
Collaborator
Istituto Di Ricerche Farmacologiche Mario Negri
Collaborator
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