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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

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

Conditions
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ALS (Amyotrophic Lateral Sclerosis), Iron Overload

Intervention / Treatment
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* Drug: Deferiprone


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 85 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Deferiprone<br>Deferiprone, 25 to 30 mg/kg per day, oral use | Drug: Deferiprone<br>* 30 mg/kg per day, oral use<br>* Other names: Ferriprox;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale |  | V3, V6, V9, V12, V15 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| Values of R2 * in MRI |  | V3, V6, V19 | 
| Oxidative stress markers analyzed blindly in blood and cerebrospinal fluid |  | V3, V9 | 
| No alteration of energy metabolism in aerobic and anaerobic blood and cerebrospinal fluid |  | V3, V9 |