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ALS (Amyotrophic Lateral Sclerosis)Phase 2SAFEFAIRALSNCT02164253

Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients

A Phase 2 study testing Deferiprone in people with ALS (Amyotrophic Lateral Sclerosis) and Iron Overload, run by University Hospital, Lille at 1 site 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 FAIR-ALS study is to investigate the safety and efficacy of a scavenger treatment of iron deferiprone, which would reduce the brain iron to limit the development of amyotrophic lateral sclerosis. It has been shown an excess of iron in the central nervous system carrying a sporadic ALS patients. Read more about this study

Who is it for?
Ages 18 to 85 years
What is being tested?
Deferiprone
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
23 participants
Where
1 siteFrance
Study dates
Sep 2013to Dec 2016
Phase
Phase 2
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 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Amyotrophic lateral sclerosis defined in accordance to the El Escorial criteria (possible, probable or defined)
  • 18 to 85 years old patient, male or female
  • Patient with social security cover

Who cannot join

  • Achieved respiratory defined by a FVC <70%
  • Evolution of more than 24 months
  • Demented subject
  • Severe malnutrition
  • Patients with treatment potentially at risk of agranulocytosis and neutropenia
  • Patients with a history of agranulocytosis or iatrogenic under haematological disease
Show all 13Show fewer
  • Incapable of giving consent
  • Indication against MRI
  • Indication against lumbar puncture
  • Patient refused lumbar puncture
  • Hypersensitivity to iron chelators
  • Concomitant treatment with antacids containing aluminum
  • Presence of another serious illness to life-threatening or disabling cons to the use of the treatment mixture of oxygen and nitrous oxide equally

Trial locations and contacts

1 site in 1 country.

France1 site

  • Hôpital Roger Salengro, CHRU de Lille

    Lille, 59000

Other trials in France

About this study

Official title: Feasibility and Safety Pilot Therapeutic Study of the Iron Chelator Deferiprone in Amyotrophic Lateral Sclerosis

The FAIR-ALS study is to investigate the safety and efficacy of a scavenger treatment of iron deferiprone, which would reduce the brain iron to limit the development of amyotrophic lateral sclerosis.

It has been shown an excess of iron in the central nervous system carrying a sporadic ALS patients. Iron overload associated with a loss of motor neurons may explain the signs of the disease (atrophy).

The investigators discuss the hypothesis that reducing excess iron, the investigators can reduce the loss of neurons and thus the progression of signs of the disease.

Read the detailed description

At the end of the study, it will propose to continue the usual quarterly patient follow up, as recommended by the French ALS centers.

Deferiprone can be administered as part of a compassionate use, for patients who want it and who do not have hypoxemia.

We therefore plan a treatment period compassionate relatively short and less than 3 years.

Being tested
Deferiprone
Medical subject headings
Amyotrophic Lateral Sclerosis, Iron Overload

What participants receive

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

  • DeferiproneDrug

    Also called Ferriprox

    30 mg/kg per day, oral use

Study groups in NCT02164253
Study groupRoleWhat they receive
DeferiproneExperimentalDeferiprone, 25 to 30 mg/kg per day, oral use
Receives: Drug: Deferiprone

What is being measured

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

  1. Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale

    Measured: V3, V6, V9, V12, V15

Secondary outcomes (8)

  1. Comparison of the progression of ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale ) score for 3 months without treatment (V0 to V3) in the period of the first three months under treatment (V3 to V6).

    Measured: SCREENING, V0, V3, V6

  2. The proportion of patients who become non-self-sufficient after 12 months with the appearance of a sub scores ALSFRS-R less than or equal to 2 on swallowing, cut food using utensils or walk.

    Measured: SCREENING, V0, V3, V6, V9, V12, V15

  3. Number of patients with anemia at 12 months defined by a hemoglobin / dL Hb <12 g

    Measured: V3, V6, V9, V12, V15

  4. Number of serious and non-serious adverse events

    Measured: SCREENING, V0, V3, V6, V9, V12, V15

  5. Progression of respiratory vital capacity tests

    Measured: SCREENING, V0, V3, V6, V9, V12, V15

  6. Values of R2 * in MRI

    Measured: V3, V6, V19

  7. Oxidative stress markers analyzed blindly in blood and cerebrospinal fluid

    Measured: V3, V9

  8. No alteration of energy metabolism in aerobic and anaerobic blood and cerebrospinal fluid

    Measured: V3, V9

Timeline

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

  1. First posted16 Jun 2014 (estimated)
  2. Study startSep 2013
  3. Primary completionDec 2016
  4. Study completionDec 2016

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
Primary purpose
Supportive care
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
23 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02164253
Sponsor's protocol ID
2012_69
Other IDs
2013-001228-21
Acronym
SAFEFAIRALS
Status last verified
Oct 2016
First submitted
20 May 2014
Last updated
20 Feb 2026
Results posted
No
Data monitoring committee
No

Publications

  • Bensimon G, Lacomblez L, Meininger V. A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group. N Engl J Med. 1994 Mar 3;330(9):585-91. doi: 10.1056/NEJM199403033300901.

    PubMed 8302340

  • Boddaert N, Le Quan Sang KH, Rotig A, Leroy-Willig A, Gallet S, Brunelle F, Sidi D, Thalabard JC, Munnich A, Cabantchik ZI. Selective iron chelation in Friedreich ataxia: biologic and clinical implications. Blood. 2007 Jul 1;110(1):401-8. doi: 10.1182/blood-2006-12-065433. Epub 2007 Mar 22.

    PubMed 17379741

  • Danzeisen R, Achsel T, Bederke U, Cozzolino M, Crosio C, Ferri A, Frenzel M, Gralla EB, Huber L, Ludolph A, Nencini M, Rotilio G, Valentine JS, Carri MT. Superoxide dismutase 1 modulates expression of transferrin receptor. J Biol Inorg Chem. 2006 Jun;11(4):489-98. doi: 10.1007/s00775-006-0099-4. Epub 2006 Apr 26.

    PubMed 16680451

  • Felice KJ. A longitudinal study comparing thenar motor unit number estimates to other quantitative tests in patients with amyotrophic lateral sclerosis. Muscle Nerve. 1997 Feb;20(2):179-85. doi: 10.1002/(sici)1097-4598(199702)20:23.0.co;2-9.

    PubMed 9040656

  • Goodall EF, Greenway MJ, van Marion I, Carroll CB, Hardiman O, Morrison KE. Association of the H63D polymorphism in the hemochromatosis gene with sporadic ALS. Neurology. 2005 Sep 27;65(6):934-7. doi: 10.1212/01.wnl.0000176032.94434.d4.

    PubMed 16186539

  • Kwiatkowski A, Ryckewaert G, Jissendi Tchofo P, Moreau C, Vuillaume I, Chinnery PF, Destee A, Defebvre L, Devos D. Long-term improvement under deferiprone in a case of neurodegeneration with brain iron accumulation. Parkinsonism Relat Disord. 2012 Jan;18(1):110-2. doi: 10.1016/j.parkreldis.2011.06.024. Epub 2011 Aug 6. No abstract available.

    PubMed 21821461

  • Kong J, Xu Z. Massive mitochondrial degeneration in motor neurons triggers the onset of amyotrophic lateral sclerosis in mice expressing a mutant SOD1. J Neurosci. 1998 May 1;18(9):3241-50. doi: 10.1523/JNEUROSCI.18-09-03241.1998.

    PubMed 9547233

  • Langkammer C, Enzinger C, Quasthoff S, Grafenauer P, Soellinger M, Fazekas F, Ropele S. Mapping of iron deposition in conjunction with assessment of nerve fiber tract integrity in amyotrophic lateral sclerosis. J Magn Reson Imaging. 2010 Jun;31(6):1339-45. doi: 10.1002/jmri.22185.

    PubMed 20512885

  • Lin J, Diamanduros A, Chowdhury SA, Scelsa S, Latov N, Sadiq SA. Specific electron transport chain abnormalities in amyotrophic lateral sclerosis. J Neurol. 2009 May;256(5):774-82. doi: 10.1007/s00415-009-5015-8. Epub 2009 Feb 25.

    PubMed 19240958

  • Miller R, Bradley W, Cudkowicz M, Hubble J, Meininger V, Mitsumoto H, Moore D, Pohlmann H, Sauer D, Silani V, Strong M, Swash M, Vernotica E; TCH346 Study Group. Phase II/III randomized trial of TCH346 in patients with ALS. Neurology. 2007 Aug 21;69(8):776-84. doi: 10.1212/01.wnl.0000269676.07319.09.

    PubMed 17709710

  • Moreau C, Gosset P, Kluza J, Brunaud-Danel V, Lassalle P, Marchetti P, Defebvre L, Destee A, Devos D. Deregulation of the hypoxia inducible factor-1alpha pathway in monocytes from sporadic amyotrophic lateral sclerosis patients. Neuroscience. 2011 Jan 13;172:110-7. doi: 10.1016/j.neuroscience.2010.10.040. Epub 2010 Oct 25.

    PubMed 20977930

  • Moreau C, Devos D, Gosset P, Brunaud-Danel V, Tonnel AB, Lassalle P, Defebvre L, Destee A. [Mechanisms of deregulated response to hypoxia in sporadic amyotrophic lateral sclerosis: a clinical study]. Rev Neurol (Paris). 2010 Mar;166(3):279-83. doi: 10.1016/j.neurol.2009.05.018. Epub 2009 Aug 5. French.

    PubMed 19660777

  • Moreau C, Gosset P, Brunaud-Danel V, Lassalle P, Degonne B, Destee A, Defebvre L, Devos D. CSF profiles of angiogenic and inflammatory factors depend on the respiratory status of ALS patients. Amyotroph Lateral Scler. 2009 Jun;10(3):175-81. doi: 10.1080/17482960802651725.

    PubMed 19177252

  • Moreau C, Devos D, Brunaud-Danel V, Defebvre L, Perez T, Destee A, Tonnel AB, Lassalle P, Just N. Paradoxical response of VEGF expression to hypoxia in CSF of patients with ALS. J Neurol Neurosurg Psychiatry. 2006 Feb;77(2):255-7. doi: 10.1136/jnnp.2005.070904.

    PubMed 16421133

  • Moreau C, Devos D, Brunaud-Danel V, Defebvre L, Perez T, Destee A, Tonnel AB, Lassalle P, Just N. Elevated IL-6 and TNF-alpha levels in patients with ALS: inflammation or hypoxia? Neurology. 2005 Dec 27;65(12):1958-60. doi: 10.1212/01.wnl.0000188907.97339.76.

    PubMed 16380619

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Deferiprone in people with ALS (Amyotrophic Lateral Sclerosis) and Iron Overload, run by University Hospital, Lille at 1 site 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 3 inclusion and 13 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 20 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this 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.

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?

Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale (time frame: V3, V6, V9, V12, V15). 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 NCT02164253, which also lists 22 linked publications.

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 20 Feb 2026.

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