Could an Iron-Removing pill slow ALS? new trial results are in
NCT ID NCT03293069
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This completed Phase 2/3 trial tested whether deferiprone, a drug that removes excess iron from the body, can slow the progression of ALS (Lou Gehrig's disease). 372 people with early-stage ALS took either deferiprone or a placebo for 12 months. The study measured changes in physical function and survival. The goal was to see if reducing iron buildup in the brain could protect nerve cells and slow the disease.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- deferiprone (an iron-removing drug)
- What this could lead to
- If it works, this could slow the progression of ALS and extend survival by removing excess iron from the brain.
- What could go wrong
- This is a mid-stage trial, so results may not confirm benefit. ALS is complex, and iron chelation might not change the disease course. Side effects from deferiprone are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2/3
Runs two stages together: whether the treatment works, then large-scale confirmation.
- Participants
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372 people
The number who actually took part.
- Started
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Jan 2019
- Finished
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May 2024
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Categorized as; possible, laboratory supported probable, probable, or definite ALS (revised El Escorial criteria) * Spinal and bulbar forms of ALS * Duration of the disease of less than 18 months since the first symptoms of motor deficit or amyotrophy (isolated cramps or fasciculation will not be considered). * Duration of the disease of less than 6 months since the diagnosis * An upright slow vital capacity ≥ 75% of the predicted value for age, height, and sex or at least one test on inspiratory pressure ≥ 60% of the predicted value for age, height, and sex which could be either maximal inspiratory pressure or a SNIFF test. (The best predictive test is sniff test but sometime patients are not able to do it.) (In case of a limit abnormal value for one of them, it will be recommended that patient re-assessment occurs three months later). * A mild functional handicap score for ALSFRS-R ≥36 * An upright slow vital capacity \> 70% of the predicted value for age, height, and sex and * Able to swallow (required for oral treatment) * Patients weight included between 40 kg and 130 kg * If the patient is under riluzole, it has to be for at least 1 month before inclusion with stable dose (to rule out the principal risk of hepatitis) Exclusion Criteria: * Patients with high frequency of comorbidity or vital risks that may reasonably impair life expectancy * Progressing axis I psychiatric disorders (psychosis, hallucinations, substance addiction, bipolar disorder, severe depression, suicidal ideation), in accordance with the Diagnostic and Statistical Manual of Mental Disorders. Before entry into the study, exclusion or stabilization of conditions must occur for patients suffering from mild or moderate depressive episodes (not in remission) or severe and uncontrolled anxiety. * Dementia according to the Diagnostic and Statistical Manual of Mental Disorders * Exposure to any other experimental drug up to 30 days before day 1 * Due to the risk of agranulocytosis (estimated at 2%) caused by the Investigational Medicinal Products (IMPs) and the unknown mechanism by which this agranulocytosis is induced, combining Deferiprone with other medicinal products known to cause agranulocytosis (as described in the IB) will not be allowed. Such medicinal products include clozapine as well as some NSAIDs (e.g. Phenylbutazone or Metamizole), antithyroid agents, sulfonamide antibiotics or methotrexate. * A history of relapsing neutropenia * Patients with agranulocytosis or with a history of agranulocytosis. * Hypersensitivity to Deferiprone * Patients with anaemia (regardless of latter aetiology) or a history of another haematological disease. Haemochromatosis is not an exclusion criterion if controlled. * Pregnant or breastfeeding women or women of childbearing potential not taking highly effective contraception. * Kidney or liver failure. * Inability to provide informed consent. * Participation in another clinical trial within 1 month prior to inclusion in the study * Patients under trusteeship
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Aphm Hopital La Timone
Marseille, France
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C H U Dupuytren Limoges
Limoges, France
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Chr Angers
Angers, France
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Chru Brest
Brest, France
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Chu Cote de Nacre - Caen
Caen, France
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Chu Toulouse
Toulouse, 31300, France
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Chu de Bordeaux - Talence
Talence, France
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Chu de Clermont-Ferrand
Clermont-Ferrand, France
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Chu de Nancy
Nancy, France
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Chu de Nice Hopital Pasteur
Nice, France
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Chu de Tours
Tours, France
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Hopital Pierre Wertheimer - Hcl - Bron
Bron, France
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Hopital de Hautepierre
Strasbourg, 67091, France
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Hu Pitie Salpetriere Aphp
Paris, 75013, France
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Hôpital Roger Salengro, CHU
Lille, 59000, France
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