Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 14 November 2022
• Main ID: NCT03293069
• Date of registration: 21/09/2017
• Prospective Registration: Yes
• Primary sponsor: University Hospital, Lille
• Public title: Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis FAIR-ALS II
• Scientific title: Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis: Multicentre, Parallel-group, Placebo-controlled, Randomized Clinical Trial of Deferiprone
• Date of first enrolment: January 1, 2019
• Target sample size: 372
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03293069
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2/Phase 3

Countries of recruitment

France

Contacts

• Name: David Devos, MD,PhD
• Affiliation: University Hospital, Lille

Key inclusion & exclusion criteria

Inclusion Criteria:

- Categorized as; possible, laboratory supported probable, probable, or definite ALS
(revised El Escorial criteria)

- Spinal and bulbar forms of ALS

- Duration of the disease of less than 18 months since the first symptoms of motor
deficit or amyotrophy (isolated cramps or fasciculation will not be considered).

- Duration of the disease of less than 6 months since the diagnosis

- An upright slow vital capacity = 75% of the predicted value for age, height, and sex
or at least one test on inspiratory pressure = 60% of the predicted value for age,
height, and sex which could be either maximal inspiratory pressure or a SNIFF test.
(The best predictive test is sniff test but sometime patients are not able to do it.)
(In case of a limit abnormal value for one of them, it will be recommended that
patient re-assessment occurs three months later).

- A mild functional handicap score for ALSFRS-R =36

- An upright slow vital capacity > 70% of the predicted value for age, height, and sex
and

- Able to swallow (required for oral treatment)

- Patients weight included between 40 kg and 130 kg

- If the patient is under riluzole, it has to be for at least 1 month before inclusion
with stable dose (to rule out the principal risk of hepatitis)

Exclusion Criteria:

- Patients with high frequency of comorbidity or vital risks that may reasonably impair
life expectancy

- Progressing axis I psychiatric disorders (psychosis, hallucinations, substance
addiction, bipolar disorder, severe depression, suicidal ideation), in accordance with
the Diagnostic and Statistical Manual of Mental Disorders. Before entry into the
study, exclusion or stabilization of conditions must occur for patients suffering from
mild or moderate depressive episodes (not in remission) or severe and uncontrolled
anxiety.

- Dementia according to the Diagnostic and Statistical Manual of Mental Disorders

- Exposure to any other experimental drug up to 30 days before day 1

- Due to the risk of agranulocytosis (estimated at 2%) caused by the Investigational
Medicinal Products (IMPs) and the unknown mechanism by which this agranulocytosis is
induced, combining Deferiprone with other medicinal products known to cause
agranulocytosis (as described in the IB) will not be allowed. Such medicinal products
include clozapine as well as some NSAIDs (e.g. Phenylbutazone or Metamizole),
antithyroid agents, sulfonamide antibiotics or methotrexate.

- A history of relapsing neutropenia

- Patients with agranulocytosis or with a history of agranulocytosis.

- Hypersensitivity to Deferiprone

- Patients with anaemia (regardless of latter aetiology) or a history of another
haematological disease. Haemochromatosis is not an exclusion criterion if controlled.

- Pregnant or breastfeeding women or women of childbearing potential not taking highly
effective contraception.

- Kidney or liver failure.

- Inability to provide informed consent.

- Participation in another clinical trial within 1 month prior to inclusion in the study

- Patients under trusteeship



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Deferiprone

Drug: Placebo Oral Tablet

Primary Outcome(s)

CAFS score (Combined Assessment of Function and Survival) [Time Frame: at 12 months]

Secondary Outcome(s)

All-cause and respiratory insufficiency mortality [Time Frame: at 12 months]

Changes in muscle strength [Time Frame: Baseline, at 12 months]

Change in Quality of life [Time Frame: Baseline, at 12 months]

Fronto-Temporal Dementia (FTD) criteria [Time Frame: at 12 months]

Change in Edinburgh Cognitive and Behavioural Amyotrophic Lateral Sclerosis Screen (ECAS) [Time Frame: Baseline, at 12 months]

Change in Montreal Cognitive Assessment (MoCA) [Time Frame: Baseline, at 12 months]

Change in the slow vital capacity [Time Frame: Baseline, at 12 months]

Changes in body weight [Time Frame: Baseline, at 12 months]

Changes in ALS Functional Rating Scale-Revised (ALSFRS-R) total score [Time Frame: Baseline, at 12 months]

DSMIV criteria [Time Frame: at 12 months]

Secondary ID(s)

2017-003763-35

2016_76

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ministry of Health, France