Iron Overload Clinical Trial
Official title:
Feasibility and Safety Pilot Therapeutic Study of the Iron Chelator Deferiprone in Amyotrophic Lateral Sclerosis
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.
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