This trial is active, not recruiting.

Conditions als (amyotrophic lateral sclerosis), iron overload
Treatment deferiprone
Phase phase 2
Sponsor University Hospital, Lille
Collaborator ApoPharma
Start date September 2013
End date December 2016
Trial size 18 participants
Trial identifier NCT02164253, 2012_69, 2013-001228-21


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose supportive care
Deferiprone, 25 to 30 mg/kg per day, oral use
deferiprone Ferriprox
30 mg/kg per day, oral use

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

Eligibility Criteria

Male or female participants from 18 years up to 85 years old.

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

Additional Information

Official title Feasibility and Safety Pilot Therapeutic Study of the Iron Chelator Deferiprone in Amyotrophic Lateral Sclerosis
Principal investigator Caroline Moreau, MD
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.
Trial information was received from ClinicalTrials.gov and was last updated in May 2016.
Information provided to ClinicalTrials.gov by University Hospital, Lille.