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

University Hospital, Lille23 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

SUPPORTIVE_CARE

Masking

NONE

Enrollment

Conditions

ALS (Amyotrophic Lateral Sclerosis)Iron Overload

Eligibility

Sex: ALLMin age: 18 YearsMax age: 85 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale

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

Secondary Outcomes

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

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

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

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

Number of serious and non-serious adverse events

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

Progression of respiratory vital capacity tests

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

Values of R2 * in MRI

Time frame: V3, V6, V19

Oxidative stress markers analyzed blindly in blood and cerebrospinal fluid

Time frame: V3, V9

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

Time frame: V3, V9

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

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes