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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
Sponsor:
Collaborator:
ApoPharma
Information provided by (Responsible Party):
University Hospital, Lille

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
ALS (Amyotrophic Lateral Sclerosis) Iron Overload Drug: Deferiprone 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 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


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 Name: Ferriprox




Primary Outcome Measures :
  1. Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale [ Time Frame: 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). [ Time Frame: 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. [ Time Frame: SCREENING, V0, V3, V6, V9, V12, V15 ]
  3. Number of patients with anemia at 12 months defined by a hemoglobin / dL Hb <12 g [ Time Frame: V3, V6, V9, V12, V15 ]
  4. Number of serious and non-serious adverse events [ Time Frame: SCREENING, V0, V3, V6, V9, V12, V15 ]
  5. Progression of respiratory vital capacity tests [ Time Frame: SCREENING, V0, V3, V6, V9, V12, V15 ]
  6. Values of R2 * in MRI [ Time Frame: V3, V6, V19 ]
  7. Oxidative stress markers analyzed blindly in blood and cerebrospinal fluid [ Time Frame: V3, V9 ]
  8. No alteration of energy metabolism in aerobic and anaerobic blood and cerebrospinal fluid [ Time Frame: V3, V9 ]


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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

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


Locations
France
Hôpital Roger Salengro, CHRU de Lille
Lille, France, 59000
Sponsors and Collaborators
University Hospital, Lille
ApoPharma
Investigators
Principal Investigator: Caroline Moreau, MD UH Lille

Publications:

Responsible Party: University Hospital, Lille
ClinicalTrials.gov Identifier: NCT02164253     History of Changes
Other Study ID Numbers: 2012_69
2013-001228-21 ( EudraCT Number )
First Posted: June 16, 2014    Key Record Dates
Last Update Posted: March 9, 2017
Last Verified: October 2016

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Deferiprone
Sclerosis
Motor Neuron Disease
Iron Overload
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Iron Metabolism Disorders
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action