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Completed

NCT Number: NCT02164253

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

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.

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Key information

Conditions

ALS (Amyotrophic Lateral Sclerosis) Amyotrophic Lateral Sclerosis Central Nervous System Diseases Iron Metabolism Disorders Iron Overload Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Age range

18 year–85 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hôpital Roger Salengro, CHRU de Lille

Lille, 59000, France

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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
  • 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

Treatment and study plan

Deferiprone

Drug

30 mg/kg per day, oral use

Other names: Ferriprox

Primary outcomes

  1. Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale

    Time frame: V3, V6, V9, V12, V15

Secondary outcomes

  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).

    Time frame: 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.

    Time frame: SCREENING, V0, V3, V6, V9, V12, V15

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

    Time frame: V3, V6, V9, V12, V15

  4. Number of serious and non-serious adverse events

    Time frame: SCREENING, V0, V3, V6, V9, V12, V15

  5. Progression of respiratory vital capacity tests

    Time frame: SCREENING, V0, V3, V6, V9, V12, V15

  6. Values of R2 * in MRI

    Time frame: V3, V6, V19

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

    Time frame: V3, V9

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

    Time frame: V3, V9

Sponsors and collaborators

Lead sponsor

University Hospital, Lille

Other

Collaborators

  • ApoPharma

Registry information

Official study title

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

Acronym: SAFEFAIRALS

Important dates

Study start
2013
Primary completion
2016
Study completion
2016
First posted
Jun 16, 2014
Registry last updated
Feb 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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