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

Hanyang University Seoul Hospital

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: recombinant human erythropoietin(rhEPO)

Study type: Interventional

Funder types: Other

Identifiers

NCT03835507

HYNR-EPO

Details and patient eligibility

About

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.

Enrollment: 64 estimated patients

Sex: All

Ages: 25 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Other

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

---

64 participants in 3 patient groups, including a placebo group

Control group

Placebo Comparator group

Description:

Inject Normal saline 100ml \* 12 times (1month apart)

Treatment:

Drug: recombinant human erythropoietin(rhEPO)

Low dose group

Experimental group

Description:

Inject 500IU/kg of EPO mixed with normal saline 100ml \* 12 times (1month apart)

Treatment:

Drug: recombinant human erythropoietin(rhEPO)

High dose group

Experimental group

Description:

Inject 750IU/kg of EPO mixed with normal saline 100ml \* 12 times (1month apart)

Treatment:

Drug: recombinant human erythropoietin(rhEPO)