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Amyotrophic Lateral SclerosisPhase 2/3FAIR-ALS IINCT03293069

Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis

A Phase 2/3 study testing Deferiprone in people with Amyotrophic Lateral Sclerosis, run by University Hospital, Lille at 15 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The alteration of iron metabolism is reported in animal models of amyotrophic lateral sclerosis (ALS) as well as in sporadic and genetic forms (SOD1 and C9orf72) of ALS. The high iron concentration of the brain, due to its high energy demand (high oxygen consumption), makes motor neurons particularly vulnerable to… Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Amyotrophic Lateral Sclerosis
What is being tested?
Deferiprone
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
372 participants
Where
15 sitesFrance
Study dates
30 Jan 2019to 6 May 2024
Phase
Phase 2/3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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)

Who cannot join

  • 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
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  • 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

Trial locations and contacts

15 sites in 1 country.

15 sites in 1 country

France15 sites

  • Chr Angers

    Angers

  • Chru Brest

    Brest

  • Hopital Pierre Wertheimer - Hcl - Bron

    Bron

  • Chu Cote de Nacre - Caen

    Caen

  • Chu de Clermont-Ferrand

    Clermont-Ferrand

  • Hôpital Roger Salengro, CHU

    Lille, 59000

  • C H U Dupuytren Limoges

    Limoges

  • Aphm Hopital La Timone

    Marseille

  • Chu de Nancy

    Nancy

  • Chu de Nice Hopital Pasteur

    Nice

  • Hu Pitie Salpetriere Aphp

    Paris, 75013

  • Hopital de Hautepierre

    Strasbourg, 67091

  • Chu de Bordeaux - Talence

    Talence

  • Chu Toulouse

    Toulouse, 31300

  • Chu de Tours

    Tours

Other trials in France

About this study

Official title: Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis: Multicentre, Parallel-group, Placebo-controlled, Randomized Clinical Trial of Deferiprone

The alteration of iron metabolism is reported in animal models of amyotrophic lateral sclerosis (ALS) as well as in sporadic and genetic forms (SOD1 and C9orf72) of ALS. The high iron concentration of the brain, due to its high energy demand (high oxygen consumption), makes motor neurons particularly vulnerable to energy deficit and oxidative stress. Post-mortem examinations and MRI scans in patients with ALS have found signs of iron accumulation in the central motor tract; and a high level of serum ferritin, which is a marker of iron levels, is associated with a lower prognosis. In ALS mouse models, the use of iron chelators has demonstrated neuroprotection and increased life expectancy, suggesting that elimination of excess iron from the brain can prevent neuronal loss and, consequently, a slow progression of the disease. Conservative chelation of iron refers to a modality whereby much of the iron that binds to the chelator is redistributed in the body rather than exhausted. Using a chelator, deferiprone, with this feature, in a safety pilot study, a very good safety profile was observed. Deferiprone eliminated excess iron from brain regions, reduced oxidative damage and cell death associated with regional iron deposits with no apparent negative impact on the iron levels needed. Now, the efficacy of this new therapeutic modality of neuroprotection is being evaluated in a randomized, double-blind, placebo-controlled, multicenter study.

Being tested
Deferiprone, Placebo Oral Tablet
Keywords
Conservative Iron Chelation, Disease-modifying Strategy
Medical subject headings
Amyotrophic Lateral Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DeferiproneDrug

    Also called DFP

    One 600 mg delayed-release tablets of deferiprone twice a day, for at 30 mg/kg/day

  • Placebo Oral TabletDrug

    the placebo twice daily morning and evening.

Study groups in NCT03293069
Study groupRoleWhat they receive
DeferiproneExperimentalHalf of participants will receive twice-daily oral deferiprone taken over 12 months.
Receives: Drug: Deferiprone
PlaceboPlacebo comparatorHalf of participants will receive the placebo Twice-daily oral placebo taken over 12 months
Receives: Drug: Placebo Oral Tablet

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. CAFS score (Combined Assessment of Function and Survival)

    CAFS score based on changes in amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) total scores and time to death from baseline (randomization visit) to 12 months

    Measured: at 12 months

Secondary outcomes (10)

  1. Changes in ALS Functional Rating Scale-Revised (ALSFRS-R) total score

    Measured: Baseline, at 12 months

  2. All-cause and respiratory insufficiency mortality

    Time to death for all cause or respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation for ≥ 23 h per day for 14 consecutive days) from baseline until 12 month

    Measured: at 12 months

  3. Changes in muscle strength

    Muscle strength measurements are determined by the overall mega-score for handheld dynamometry and manual muscular testing with a validated medical device provided

    Measured: Baseline, at 12 months

  4. Change in the slow vital capacity

    The slow vital capacity is measure by the maximum amount of air that can be exhaled following a deep breath. Reflecting the Respiratory insufficiency.

    Measured: Baseline, at 12 months

  5. Changes in body weight

    Measured: Baseline, at 12 months

  6. Change in Quality of life

    Quality of life assessed using the five-item form of the ALS assessment questionnaire (ALSAQ-5) from baseline to 12 months

    Measured: Baseline, at 12 months

  7. DSMIV criteria

    Dementia (yes/no)

    Measured: at 12 months

  8. Fronto-Temporal Dementia (FTD) criteria

    Using the revised guidelines for the diagnosis of behavioral variant frontotemporal dementia based on recent literature and collective experience by Rascovsky K et al 2011; Lamarre AK et al 2013

    Measured: at 12 months

  9. Change in Montreal Cognitive Assessment (MoCA)

    MoCA evaluated of mild cognitive dysfunction.

    Measured: Baseline, at 12 months

  10. Change in Edinburgh Cognitive and Behavioural Amyotrophic Lateral Sclerosis Screen (ECAS)

    ECAS determine cognitive and behavioral changes of patients suffering from Amyotrophic Lateral Sclerosis.

    With ECAS, ALS-specific (fluency, executive functions and social cognition, language) and ALS-nonspecific (memory, visuospatial functions) functions can be analyzed to enable the distinction from other diseases with cognitive and behavioral impairments.

    Measured: Baseline, at 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Sep 2017
  2. Study start30 Jan 2019
  3. Primary completion11 Nov 2023
  4. Study completion6 May 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
372 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03293069
Sponsor's protocol ID
2016_76
Other IDs
2017-003763-35, 2017
Acronym
FAIR-ALS II
Status last verified
Dec 2025
First submitted
21 Sep 2017
Last updated
5 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Saini A, Chawla PA. Breaking barriers with tofersen: Enhancing therapeutic opportunities in amyotrophic lateral sclerosis. Eur J Neurol. 2024 Feb;31(2):e16140. doi: 10.1111/ene.16140. Epub 2023 Nov 17.

    PubMed 37975798

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Deferiprone in people with Amyotrophic Lateral Sclerosis, run by University Hospital, Lille at 15 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

CAFS score (Combined Assessment of Function and Survival) (time frame: at 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03293069, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Dec 2025.

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