Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients (SAFEFAIRALS)
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|ClinicalTrials.gov Identifier: NCT02164253|
Recruitment Status : Completed
First Posted : June 16, 2014
Last Update Posted : March 9, 2017
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.
|Condition or disease||Intervention/treatment||Phase|
|ALS (Amyotrophic Lateral Sclerosis) Iron Overload||Drug: Deferiprone||Phase 2|
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 (Clinical Trial)|
|Actual Enrollment :||23 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Primary Purpose:||Supportive Care|
|Official Title:||Feasibility and Safety Pilot Therapeutic Study of the Iron Chelator Deferiprone in Amyotrophic Lateral Sclerosis|
|Study Start Date :||September 2013|
|Actual Primary Completion Date :||December 2016|
|Actual Study Completion Date :||December 2016|
Deferiprone, 25 to 30 mg/kg per day, oral use
30 mg/kg per day, oral use
Other Name: 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02164253
|Hôpital Roger Salengro, CHRU de Lille|
|Lille, France, 59000|
|Principal Investigator:||Caroline Moreau, MD||UH Lille|