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

NCT02164253 | Completed Phase 2

• 2 other identifiers: 2012_692013-001228-21
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

ALS (Amyotrophic Lateral Sclerosis); Iron Overload

Outcome Measures

Primary Outcomes (1)

• Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale

  V3, V6, V9, V12, V15

Secondary Outcomes (8)

• 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).

  SCREENING, V0, V3, V6

• 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.

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

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

  V3, V6, V9, V12, V15

• Number of serious and non-serious adverse events

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

• Progression of respiratory vital capacity tests

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

Study Arms (1)

Deferiprone

EXPERIMENTAL

Deferiprone, 25 to 30 mg/kg per day, oral use

Drug: Deferiprone

Interventions

Deferiprone DRUG

30 mg/kg per day, oral use

Also known as: Ferriprox

Deferiprone

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• University Hospital, Lille: lead
• ApoPharma: collaborator

Study Sites (1)

Hôpital Roger Salengro, CHRU de Lille

Lille, 59000, France

Location

Related Publications (22)

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• Moreau C, Danel V, Devedjian JC, Grolez G, Timmerman K, Laloux C, Petrault M, Gouel F, Jonneaux A, Dutheil M, Lachaud C, Lopes R, Kuchcinski G, Auger F, Kyheng M, Duhamel A, Perez T, Pradat PF, Blasco H, Veyrat-Durebex C, Corcia P, Oeckl P, Otto M, Dupuis L, Garcon G, Defebvre L, Cabantchik ZI, Duce J, Bordet R, Devos D. Could Conservative Iron Chelation Lead to Neuroprotection in Amyotrophic Lateral Sclerosis? Antioxid Redox Signal. 2018 Sep 10;29(8):742-748. doi: 10.1089/ars.2017.7493. Epub 2018 Feb 8.

  PMID: 29287521 RESULT

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Iron Overload

Interventions

Deferiprone

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Iron Metabolism Disorders

Intervention Hierarchy (Ancestors)

Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Caroline Moreau, MD

  UH Lille

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 20, 2014
• First Posted: June 16, 2014
• Study Start: September 1, 2013
• Primary Completion: December 1, 2016
• Study Completion: December 1, 2016
• Last Updated: February 20, 2026

Record last verified: 2016-10

Locations

FR (1)