Status:
COMPLETED
Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis
Lead Sponsor:
University Hospital, Lille
Collaborating Sponsors:
Ministry of Health, France
Conditions:
Amyotrophic Lateral Sclerosis
Eligibility:
All Genders
18-75 years
Phase:
PHASE2
PHASE3
Brief Summary
The alteration of iron metabolism is reported in animal models of amyotrophic lateral sclerosis (ALS) as well as in sporadic and genetic forms (SOD1 and C9orf72) of ALS. The high iron concentration of...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
January 30 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 6 2024
Estimated Enrollment :
372 Patients enrolled
Trial Details
Trial ID
NCT03293069
Start Date
January 30 2019
End Date
May 6 2024
Last Update
December 5 2025
Active Locations (15)
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1
Chr Angers
Angers, France
2
Chru Brest
Brest, France
3
Hopital Pierre Wertheimer - Hcl - Bron
Bron, France
4
Chu Cote de Nacre - Caen
Caen, France