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

University Hospital, Lille

Status and phase: Completed; Phase 2

Conditions: Iron Overload; ALS (Amyotrophic Lateral Sclerosis)

Treatments: Drug: Deferiprone

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02164253

2013-001228-21 (EudraCT Number)

2012_69

Details and patient eligibility

About

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.

Full 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.

Enrollment: 23 patients

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Supportive Care

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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23 participants in 1 patient group

Deferiprone

Experimental group

Description:

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

Treatment:

Drug: Deferiprone