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

Sponsor
University Hospital, Lille (Other)
Overall Status
Completed
CT.gov ID
NCT02164253
Collaborator
ApoPharma (Industry)
23
Enrollment
1
Location
1
Arm
39
Duration (Months)
0.6
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase
Phase 2

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.

Study Design

Study Type:
Interventional
Actual Enrollment :
23 participants
Allocation:
N/A
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 :
Sep 1, 2013
Actual Primary Completion Date :
Dec 1, 2016
Actual Study Completion Date :
Dec 1, 2016

Arms and Interventions

Arm Intervention/Treatment
Experimental: Deferiprone

Deferiprone, 25 to 30 mg/kg per day, oral use

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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale [V3, V6, V9, V12, V15]

    Secondary Outcome Measures

    1. 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]

    2. 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]

    3. Number of patients with anemia at 12 months defined by a hemoglobin / dL Hb <12 g [V3, V6, V9, V12, V15]

    4. Number of serious and non-serious adverse events [SCREENING, V0, V3, V6, V9, V12, V15]

    5. Progression of respiratory vital capacity tests [SCREENING, V0, V3, V6, V9, V12, V15]

    6. Values of R2 * in MRI [V3, V6, V19]

    7. Oxidative stress markers analyzed blindly in blood and cerebrospinal fluid [V3, V9]

    8. No alteration of energy metabolism in aerobic and anaerobic blood and cerebrospinal fluid [V3, V9]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 85 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Hôpital Roger Salengro, CHRU de Lille Lille France 59000

    Sponsors and Collaborators

    • University Hospital, Lille
    • ApoPharma

    Investigators

    • Principal Investigator: Caroline Moreau, MD, UH Lille

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    Responsible Party:
    University Hospital, Lille
    ClinicalTrials.gov Identifier:
    NCT02164253
    Other Study ID Numbers:
    • 2012_69
    • 2013-001228-21
    First Posted:
    Jun 16, 2014
    Last Update Posted:
    Mar 9, 2017
    Last Verified:
    Oct 1, 2016
    Additional relevant MeSH terms:
    Motor Neuron Disease
    Amyotrophic Lateral Sclerosis
    Iron Overload
    Sclerosis
    Deferiprone

    Study Results

    No Results Posted as of Mar 9, 2017