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

Iron Overload Clinical Trial

— SAFEFAIRALS  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02164253
• Other study ID #: 2012_69
• Secondary ID: 2013-001228-21
• Status: Completed
• Phase: Phase 2
• First received: May 20, 2014
• Last updated: March 8, 2017
• Start date: September 2013
• Est. completion date: December 2016

Study information

• Verified date: October 2016
• Source: University Hospital, Lille
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 23
• Est. completion date: December 2016
• Est. primary completion date: December 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 85 Years

Eligibility

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

Related Conditions & MeSH terms

• ALS (Amyotrophic Lateral Sclerosis)
• Amyotrophic Lateral Sclerosis
• Iron Overload
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• Deferiprone
30 mg/kg per day, oral use

Locations

Country	Name	City	State
France	Hôpital Roger Salengro, CHRU de Lille	Lille

Sponsors (2)

Lead Sponsor	Collaborator
University Hospital, Lille	ApoPharma

Country where clinical trial is conducted

France, 

References & Publications (21)

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* Note: There are 21 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale		V3, V6, V9, V12, V15
Secondary	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).		SCREENING, V0, V3, V6
Secondary	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.		SCREENING, V0, V3, V6, V9, V12, V15
Secondary	Number of patients with anemia at 12 months defined by a hemoglobin / dL Hb <12 g		V3, V6, V9, V12, V15
Secondary	Number of serious and non-serious adverse events		SCREENING, V0, V3, V6, V9, V12, V15
Secondary	Progression of respiratory vital capacity tests		SCREENING, V0, V3, V6, V9, V12, V15
Secondary	Values of R2 * in MRI		V3, V6, V19
Secondary	Oxidative stress markers analyzed blindly in blood and cerebrospinal fluid		V3, V9
Secondary	No alteration of energy metabolism in aerobic and anaerobic blood and cerebrospinal fluid		V3, V9