Clinical trial NCT03835507: Randomized, Double-blind, Safety and Efficacy of Recombinant Human Erythropoietin in Amyotrophic Lateral Sclerosis
Official title: Randomized, Double-blind, Safety and Efficacy of Recombinant Human Erythropoietin in Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Hanyang University Seoul Hospital
Sponsor class: OTHER
First posted: 2019-02-08
Enrollment: 64
Sex: ALL
Minimum age (years): 25
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

Brief summary: 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.


Eligibility 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

Primary outcomes:
measure: changes of ALSFRS-R score
timeFrame: Last visit [15th visit (15 months)]
Secondary outcomes:
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Hanyang Medical Center
Study officials:
References:
citation: 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.
citation: 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.