Colchicine Effect on Amyotrophic Lateral Sclerosis Patients

Summary

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.

Timeline

Start

2026-10

Primary completion

2028-05

Completion

2028-12

Publications

• Background Mandrioli J, Crippa V, Cereda C, Bonetto V, Zucchi E, Gessani A, Ceroni M, Chio A, D'Amico R, Monsurro MR, Riva N, Sabatelli M, Silani V, Simone IL, Soraru G, Provenzani A, D'Agostino VG, Carra S, Poletti A. Proteostasis and ALS: protocol for a phase II, randomised, double-blind, placebo-controlled, multicentre clinical trial for colchicine in ALS (Co-ALS). BMJ Open. 2019 May 30;9(5):e028486. doi: 10.1136/bmjopen-2018-028486.
• Background Gianferrari G, Cuoghi Costantini R, Crippa V, Carra S, Bonetto V, Pansarasa O, Cereda C, Zucchi E, Martinelli I, Simonini C, Vicini R, Fini N, Trojsi F, Passaniti C, Ticozzi N, Doretti A, Diamanti L, Fiamingo G, Conte A, Dalla Bella E, D'Errico E, Scarian E, Pasetto L, Antoniani F, Galli V, Casarotto E; Co-ALS Investigators Group; D'Amico R, Poletti A, Mandrioli J. Colchicine treatment in amyotrophic lateral sclerosis: safety, biological and clinical effects in a randomized clinical trial. Brain Commun. 2024 Sep 5;6(5):fcae304. doi: 10.1093/braincomms/fcae304. eCollection 2024.

Drugs

Indications