Randomized, Double-blind, Safety and Efficacy of Recombinant Human Erythropoietin in Amyotrophic Lateral Sclerosis

Study Overview
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Brief Summary
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Erythropoietin is neuroprotective in animal models of neurodegenerative diseases including amyotrophic lateral sclerosis (ALS). The aim of this study was to determine the safety and feasibility of repetitive high-dose recombinant human erythropoietin (rhEPO) therapy in ALS patients.

Official Title
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Randomized, Double-blind, Safety and Efficacy of Recombinant Human Erythropoietin in Amyotrophic Lateral Sclerosis

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: recombinant human erythropoietin(rhEPO)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age between 25 to 80 upper motor neuron signs and lower motor neuron signs were identified in neurological examination. Meet the revised El Escorial Criteria for clinically possible, probable-laboratory -supported, probable, definite ALS. Disease duration < 3 years (Within 3 years from symptom onset) ALSFRS-R score between 21 to 46 Patient who can visit an outpatient under the aid of his or her own walking or caregivers. The person who have agreed in writing to participate in this clinical trial by themselves and the legal representative FVC over 50% at screening Exclusion Criteria: Person who were not compatible with ALS Patient with PLS or PMA A group of patients who are concerned about the adverse effects of the drug administration (e.g. malignant hypertension,...) ALSFRS-R score below 20 at screening Ventilator user or Tracheostomy state patients at screening Gastrostomy state at screening FVC below 50% at screening or patient who cannot perform FVC test. EKG abnormality, history of coronary stent , CABG at screening Person who was given another clinical trial drug three months prior to screening. History of seizure/ epilepsy Abnormal renal function (serem creatinine > 2.0mg/dl) Abnormal liver function(AST/ALT/bilirubin over 2 times the upper normal limit Pregnant Bleeding tendency at screening Infectious disease at screening Drug sensitivity Person who injected erythropoietin 6 months prior to screening Malignant tumor Other neurological disease (stroke, parkinson's disease, dementia...) Psychological disease Hb more than 16g/dL

Ages Eligible for Study
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Minimum Age: 25 Years
Maximum Age: 80 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: Other
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Control group<br>Inject Normal saline 100ml * 12 times (1month apart) | Drug: recombinant human erythropoietin(rhEPO)<br>* Injection of erythropoietin every months (total 12 months)<br>|
| Experimental: Low dose group<br>Inject 500IU/kg of EPO mixed with normal saline 100ml * 12 times (1month apart) | Drug: recombinant human erythropoietin(rhEPO)<br>* Injection of erythropoietin every months (total 12 months)<br>|
| Experimental: High dose group<br>Inject 750IU/kg of EPO mixed with normal saline 100ml * 12 times (1month apart) | Drug: recombinant human erythropoietin(rhEPO)<br>* Injection of erythropoietin every months (total 12 months)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| changes of ALSFRS-R score | changes between score of ALSFRS-R at initial and study end point | Last visit [15th visit (15 months)] |