NCT02164253

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 23 (actual)
• Sponsor: University Hospital, Lille · Academic / Other
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

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

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.

Conditions

• ALS (Amyotrophic Lateral Sclerosis)
• Iron Overload

Interventions

Type	Name	Description
DRUG	Deferiprone	30 mg/kg per day, oral use

Timeline

• Start date: 2013-09-01
• Primary completion: 2016-12-01
• Completion: 2016-12-01
• First posted: 2014-06-16
• Last updated: 2026-02-20

Locations

1 site across 1 country: France