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

Primary Purpose

ALS (Amyotrophic Lateral Sclerosis), Iron Overload

Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Deferiprone
Sponsored by
University Hospital, Lille
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for ALS (Amyotrophic Lateral Sclerosis)

Eligibility Criteria

18 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Hôpital Roger Salengro, CHRU de Lille

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Deferiprone

Arm Description

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

Outcomes

Primary Outcome Measures

Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale

Secondary Outcome Measures

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).
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.
Number of patients with anemia at 12 months defined by a hemoglobin / dL Hb <12 g
Number of serious and non-serious adverse events
Progression of respiratory vital capacity tests
Values of R2 * in MRI
Oxidative stress markers analyzed blindly in blood and cerebrospinal fluid
No alteration of energy metabolism in aerobic and anaerobic blood and cerebrospinal fluid

Full Information

First Posted
May 20, 2014
Last Updated
March 8, 2017
Sponsor
University Hospital, Lille
Collaborators
ApoPharma
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1. Study Identification

Unique Protocol Identification Number
NCT02164253
Brief Title
Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients
Acronym
SAFEFAIRALS
Official Title
Feasibility and Safety Pilot Therapeutic Study of the Iron Chelator Deferiprone in Amyotrophic Lateral Sclerosis
Study Type
Interventional

2. Study Status

Record Verification Date
October 2016
Overall Recruitment Status
Completed
Study Start Date
September 2013 (undefined)
Primary Completion Date
December 2016 (Actual)
Study Completion Date
December 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Lille
Collaborators
ApoPharma

4. Oversight

Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
ALS (Amyotrophic Lateral Sclerosis), Iron Overload

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
23 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Deferiprone
Arm Type
Experimental
Arm Description
Deferiprone, 25 to 30 mg/kg per day, oral use
Intervention Type
Drug
Intervention Name(s)
Deferiprone
Other Intervention Name(s)
Ferriprox
Intervention Description
30 mg/kg per day, oral use
Primary Outcome Measure Information:
Title
Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale
Time Frame
V3, V6, V9, V12, V15
Secondary Outcome Measure Information:
Title
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
Title
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
Title
Number of patients with anemia at 12 months defined by a hemoglobin / dL Hb <12 g
Time Frame
V3, V6, V9, V12, V15
Title
Number of serious and non-serious adverse events
Time Frame
SCREENING, V0, V3, V6, V9, V12, V15
Title
Progression of respiratory vital capacity tests
Time Frame
SCREENING, V0, V3, V6, V9, V12, V15
Title
Values of R2 * in MRI
Time Frame
V3, V6, V19
Title
Oxidative stress markers analyzed blindly in blood and cerebrospinal fluid
Time Frame
V3, V9
Title
No alteration of energy metabolism in aerobic and anaerobic blood and cerebrospinal fluid
Time Frame
V3, V9

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Caroline Moreau, MD
Organizational Affiliation
UH Lille
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hôpital Roger Salengro, CHRU de Lille
City
Lille
ZIP/Postal Code
59000
Country
France

12. IPD Sharing Statement

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

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