Treatment Effect of Edaravone in Patients With Amyotrophic Lateral Sclerosis (ALS)

Study Overview
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Brief Summary
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Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease that causes the death of 30,000 affected individual every year. Complex nature and unknown pathogenesis of this disease are 2 major reasons for failure of therapeutic interventions. Edaravone is a free radical scavenger that slows down functional decline and prevents from disease progression in ALS patients. FDA newly approved this drug in these patients (2017/5/5). In this study, investigators aimed to assess the treatment effect of this newly approved drug in patients with ALS in a representative Iranian population.

Official Title
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Treatment Effect of Edaravone in Patients With Amyotrophic Lateral Sclerosis (ALS) in a Representative Iranian Population

Conditions
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Neuromuscular Diseases

Intervention / Treatment
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* Drug: Edaravone
* Drug: Riluzole


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients diagnosed as definite or probable ALS according to El Escorial Criteria. ALS patients who are graded as mild or moderate according to ALS Health State Scale. Forced vital capacity of at least 80% Desire of the patient to participate in this study and Signing Written Informed Consent. Exclusion Criteria: Incidence of drug's side effects that requires discontinuation of the drug (Edaravone's side effects: Acute kidney injury, Acute allergic reactions, DIC, Thrombocytopenia, Leukopenia). Desire of the patient to discontinue participating in this study. the patient starts another drug or herb for ALS during the study.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Case group<br>ALS patients who receive the usual treatment option (Riluzole) for this disease and Edaravone. Instructions: Tab. Rilutek 50 mg PO q12hr on empty stomach. Amp. Edaravone 60 mg per day IV infusion (in normal saline during 1 hour) for 14 days in the first 28 day cycle. Amp. Edaravone 60 mg per day IV infusion (in normal saline during 1 hour) for 10 days in the following 28 day cycles after the first cycle (for 11 cycles). | Drug: Edaravone<br>* Edaravone is a free radical scavenger. this drug showed desirable effects like slowing decline of physical function by 33 percent in previous studies.<br>* Other names: RADICAVA;Drug: Riluzole<br>* Riluzole is a treatment option for amyotrophic lateral sclerosis. The occurrence of ventilator-dependence or tracheostomy is delayed in selected patients who treated with this drug.<br>* Other names: Teglutik;|
| Active Comparator: Control group<br>ALS patients who receive the usual treatment option (Riluzole) for this disease. Instructions: 1. Tab. Rilutek 50 mg PO q12hr on empty stomach. | Drug: Riluzole<br>* Riluzole is a treatment option for amyotrophic lateral sclerosis. The occurrence of ventilator-dependence or tracheostomy is delayed in selected patients who treated with this drug.<br>* Other names: Teglutik;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Functional evaluation of patient's muscle strength. | Manual Muscle Testing (MMT) will be used to evaluate functional muscle strength. This procedure evaluates the strength of some proximal and distal muscles of each limb and also the neck region. | At the time of enrolling the patient to study, and then every 3 moths in the following period of 1 year. | 
| functional status of the patient. | Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) will be used to evaluate functional status of the patient. | At the time of enrolling the patient to study, and then every 3 moths in the following period of 1 year. | 
| Quality of life in the patients | Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) will be used to assess Quality of life in the patients. The Persian version of this questionare will be used in this study. | At the time of enrolling the patient to study, and then every 3 moths in the following period of 1 year. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Amyotrophic Lateral Sclerosis, Edaravone