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

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.

Study Overview

• Status: Recruiting
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: recombinant human erythropoietin(rhEPO)

• Study Type: Interventional
• Enrollment (Anticipated): 64
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Jinseok Park, MD; Phone Number: +82-2-2290-8367; Email: jinseok.park0@gmail.com

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Recruiting
    • Hanyang medical center
    • Contact: Jinseok Park, MD; Phone Number: +82-2-2290-8367; Email: jinseok.park0@gmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 25 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

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)]

Collaborators and Investigators

• Sponsor: Hanyang University Seoul Hospital

Publications and helpful links

General Publications

• Kim HY, Moon C, Kim KS, Oh KW, Oh SI, Kim J, Kim SH. Recombinant human erythropoietin in amyotrophic lateral sclerosis: a pilot study of safety and feasibility. J Clin Neurol. 2014 Oct;10(4):342-7. doi: 10.3988/jcn.2014.10.4.342. Epub 2014 Oct 6.
• Noh MY, Cho KA, Kim H, Kim SM, Kim SH. Erythropoietin modulates the immune-inflammatory response of a SOD1(G93A) transgenic mouse model of amyotrophic lateral sclerosis (ALS). Neurosci Lett. 2014 Jun 27;574:53-8. doi: 10.1016/j.neulet.2014.05.001. Epub 2014 May 10.

Study record dates

Study Major Dates

• Study Start (Actual): June 20, 2016
• Primary Completion (Anticipated): December 1, 2020
• Study Completion (Anticipated): December 1, 2025

Study Registration Dates

• First Submitted: August 8, 2018
• First Submitted That Met QC Criteria: February 6, 2019
• First Posted (Actual): February 8, 2019

Study Record Updates

• Last Update Posted (Actual): February 8, 2019
• Last Update Submitted That Met QC Criteria: February 6, 2019
• Last Verified: February 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Hematinics; Epoetin Alfa
• Other Study ID Numbers: HYNR-EPO

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No