Testing a new treatment for ALS using erythropoietin

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

Phase1; Phase2 Interventional Hanyang University Seoul Hospital · NCT03835507

This study is testing whether a new treatment using erythropoietin can help people with early-stage ALS feel better and stay healthier.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment64 (estimated)
Ages25 Years to 80 Years
SexAll
SponsorHanyang University Seoul Hospital Academic / other
Locations1 site (Seoul)
Trial IDNCT03835507 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of recombinant human erythropoietin (rhEPO) in patients with amyotrophic lateral sclerosis (ALS). It is a randomized, double-blind study that aims to determine the feasibility of high-dose rhEPO therapy in ALS patients who meet specific eligibility criteria. Participants will be monitored for safety and potential benefits over the course of the trial. The study focuses on individuals with early-stage ALS, specifically those with a disease duration of less than three years.

Who should consider this trial

Good fit: Ideal candidates are adults aged 25 to 80 with early-stage ALS and specific neurological signs.

Not a fit: Patients with advanced ALS, those requiring ventilatory support, or with significant comorbidities may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with ALS, potentially slowing disease progression.

How similar studies have performed: Previous studies have shown neuroprotective effects of erythropoietin in animal models of ALS, but this approach in humans is still being evaluated.

Eligibility criteria

Show full inclusion / exclusion criteria
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

Where this trial is running

Seoul

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Amyotrophic Lateral Sclerosis
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.