A Phase 2 interventional study of Deferiprone in ALS (Amyotrophic Lateral Sclerosis) and Iron Overload, sponsored by University Hospital, Lille. Completed at 1 site in France. Open to participants aged 18 Years to 85 Years. Per ClinicalTrials.gov, last updated 2026-02-20.
Sponsored by University Hospital, Lille · Phase 2, Interventional, and Supportive care
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.
Exclusion Criteria:
Deferiprone, 25 to 30 mg/kg per day, oral use
Drug: Deferiprone
30 mg/kg per day, oral use
Also known as: Ferriprox
Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale
Time frame: V3, V6, V9, V12, V15
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
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Amyotrophic Lateral Sclerosis→
University Hospital, Lille