Randomized Placebo‑Controlled Trial of Colchicine to Slow Disease Progression in Adults with Amyotrophic Lateral Sclerosis

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What is this study about?

The study focuses on Amyotrophic Lateral Sclerosis, a rare condition that gradually weakens the muscles used for walking, speaking, breathing and other everyday activities. The medication being tested is a low‑dose tablet of colchicine, an oral drug that may affect the processes that lead to nerve and muscle damage. Some participants will receive a matching tablet that contains no active ingredient, referred to as a placebo.

The purpose of the trial is to find out whether taking colchicine each day can slow the typical decline in muscle function compared with the placebo.

Participants will be randomly assigned to receive either the colchicine tablet or the placebo tablet for about 30 weeks, followed by an optional open‑label period where all may receive the active drug. During the study, they will attend regular clinic visits where basic health checks, breathing tests, and questionnaires about daily abilities and quality of life are completed. The study ends after a total of roughly 66 weeks, at which point the collected information will be used to evaluate the medication’s effect.

1 randomization

after joining the study you are randomly assigned to receive either colchicine tablets or identical placebo tablets. you will not know which one you receive.

2 receive study medication

you will be given a bottle of tablets. each tablet contains 0.5 mg of colchicine or no active ingredient (placebo). the prescribed dose is 0.005 mg per kilogram of body weight per day, taken by mouth.

the exact number of tablets you need each day is calculated from your weight. for example, a person weighing 70 kg would need 0.35 mg per day, which corresponds to taking a portion of a 0.5 mg tablet as instructed by the study staff.

3 start daily medication

take the tablet once a day at the same time each day. swallow the tablet with water. the tablet may be taken with or without food, unless you are told otherwise.

4 baseline assessments

before the first dose, a series of measurements is performed:

alsfrs‑r questionnaire to record functional abilities,

roads questionnaire to assess overall disability,

forced vital capacity (fvc) breathing test,

alsaq‑40 questionnaire to evaluate quality of life.

5 regular follow‑up visits (double‑blind phase)

you will attend clinic visits at weeks 4, 8, 12, 18, 24 and 30.

at each visit the same set of assessments as at baseline is repeated: the alsfrs‑r score, roads score, fvc measurement, and the alsaq‑40 questionnaire.

these visits allow the study to compare the monthly change in scores between the colchicine and placebo groups.

6 end of double‑blind treatment

after the week 30 visit the blinded medication period ends. you will stop receiving the study tablet that was assigned during randomization.

7 open‑label extension (weeks 30‑66)

following week 30 you enter an open‑label period lasting 36 weeks, until week 66.

during this phase all participants receive colchicine at the same dose of 0.005 mg/kg/day, regardless of the original assignment.

you will continue to take the medication daily as instructed.

8 follow‑up visits (open‑label phase)

additional clinic visits are scheduled at weeks 42, 54 and 66.

the same assessments are performed at each visit: alsfrs‑r, roads, fvc, and alsaq‑40.

9 final assessment

at week 66 a final set of measurements is taken, completing the 66‑week study period.

the data collected includes all functional scores, breathing capacity, and quality‑of‑life questionnaire results.

10 survival monitoring

throughout the entire 66‑week period the study records the time from randomization to death or tracheostomy, to evaluate overall survival.

Who Can Join the Study?

Be diagnosed with amyotrophic lateral sclerosis (ALS) that is classified as probable laboratory‑supported, clinically probable, or definite according to the Revised El Escorial criteria (a set of guidelines doctors use to confirm ALS), including both sporadic and familial types.
Be a male or female aged between 18 and 80 years.
Have had the first signs of muscle weakness in the arms, legs, or speech/swallowing (known as bulbar symptoms) for 18 months or less at the time of screening.
Take a stable dose of the medication Riluzole (50 mg taken twice each day) for at least 30 days before the screening visit.
Have a body mass index ( BMI ) greater than 17.5 kg/m², which is a measure of body weight relative to height.
Show a forced vital capacity (FVC) that is at least 60 % of the normal value predicted for your gender, height, and age, indicating adequate breathing function.
Be able to give informed consent (understand the study and agree to take part) and be willing to follow all study procedures.
Use a highly effective method of contraception (birth control) if you are capable of becoming pregnant, and for male participants, also use effective contraception.

Who Cannot Join the Study?

If you have previously taken colchicine or are allergic to it.
If you are a woman who is currently pregnant (carrying a baby) or breastfeeding (feeding a baby with breast milk).
If you have participated in another randomized controlled trial (research study testing a new treatment) within the past 30 days.
If you regularly use colchicine or other strong anti‑inflammatory medicines such as corticosteroids, methotrexate, IL‑1β antagonists, or TNF‑α inhibitors.
If you are taking foods or medicines that can raise colchicine levels in your blood, such as strong CYP3A4 inhibitors (drugs that block a liver enzyme that normally helps break down medicines).
If you have ongoing inflammatory or autoimmune disorders (conditions where the immune system attacks the body), long‑lasting infections, or malignancy (cancer).
If you have severe kidney or liver failure, serious blood problems, a low white blood cell count (<4,000 cells per microliter), low platelet count (<100,000 per microliter), or low hematocrit (<30%, meaning a low proportion of red blood cells).
If you have serious other health issues such as severe heart disease, kidney or liver failure, autoimmune diseases, or any type of interstitial lung disease (lung conditions that cause scarring), as judged by the doctor.
If your ALSFRS‑R (ALS Functional Rating Scale – Revised) total score is less than 28, indicating greater disability.
If you require invasive ventilation (a breathing tube placed inside the body) or use non‑invasive ventilation (mask‑type breathing support) for more than 8 hours each day.
If you have rare inherited problems that affect sugar processing, such as galactose intolerance, lactase deficiency, or glucose‑galactose malabsorption.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Azienda Ospedaliera Universitaria Universita’ Degli Studi Della Campania Luigi Vanvitelli	Naples	Italy

Other Sites

Site Name	City	Country
Azienda Ospedaliero-Universitaria Maggiore Della Carita	Novara	Italy
Fondazione Istituto Neurologico Nazionale Casimiro Mondino	Pavia	Italy
AOU di Modena Nuovo Ospedale Civile S. Agostino Estense di Modena- Ospedale di Baggiovara	Modena	Italy
Ctoifh Cxkfxiq Nrbx	Milan	Italy

Trial status

Country	Status	Recruitment Start
Italy	Not yet recruiting	01.09.2026

Trial locations

Investigated Drugs:

Colchicine

colchicine is an oral medication that is being tested to see if it can slow the progression of amyotrophic lateral sclerosis (ALS). In this study, participants take tablets containing colchicine each day. The researchers want to find out whether regular use of this drug can help keep the disease from getting worse compared with taking no active medicine.

Investigated Diseases:

Amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis – a neurodegenerative disease that weakens the motor nerves controlling voluntary muscles. It usually starts with subtle muscle twitching or difficulty lifting objects and then spreads to other limbs. As the nerves degenerate, speaking, swallowing, and breathing muscles become progressively weaker. The loss of muscle strength continues over months to years, leading to increasing disability.

Trial ID:

2026-526460-20-00

Protocol code:

CO-ALS II

Trial Phase:

Therapeutic exploratory (Phase II)

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Randomized Placebo‑Controlled Trial of Colchicine to Slow Disease Progression in Adults with Amyotrophic Lateral Sclerosis

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?