Clinical trial of neuroEPO in mild‐to‐moderate Alzheimer’s disease

Abstract

AbstractBackgroundNeuroEPO, a nasal pharmaceutical solution of recombinant human erythropoietin with low sialic acid, was in clinical development for the treatment of mild‐moderate Alzheimer’s disease.MethodA double‐blind, randomized, placebo‐controlled, multicenter, prospective, with adaptive design, phase II/III clinical trial involving 174 patients with mild‐to‐moderate Alzheimer’s, was conducted. NeuroEPO or placebo, with different doses, was administered by nasal route 3 times a weeks for 48 weeks. The primary outcome measure was score on the 11‐item cognitive subscale of the Alzheimer’s disease Assessment Scale (ADAS‐cog11, with scores ranging from 0 to 70 and higher scores indicating greater impairment). Secondary outcome measures included Clinician’s Interview‐Based Impression of Change Plus Caregiver Input (CIBIC‐plus), Global Dementia Scale (GDS), activities of daily living (ADL), hippocampal volume change over time (measured with MRI), neuropsychological scales, electroencephalography (EEG) and adverse events.ResultThere were statistically significant differences between neuroEPO and placebo in median change from baseline in the primary outcome at week 48. The change in ADAS‐cog11 scores final‐initial (PP population) for patients in the neuroEPO 0.5 mg (n= 50, median change: ‐ 4.0), neuroEPO 1.0 mg (n=49, median change: ‐ 5.0) and placebo (n=49, median change: 4.0) groups. A difference of 8.0 points CI 95% (6.0; 10.0) neuroEPO 0.5 mg vs. placebo and 9.0 points CI 95% (6.8; 11.2) neuroEPO 1.0 mg vs. placebo at week 48, (p=0.000), was observed. Patients receiving both doses of neuroEPO also showed a significant difference in global improvement (CIBIC‐Plus, p=0.000) and electroencephalography (EEG, p=0.003) compared with placebo group. About hippocampal volume change, 105 patients were analyzed. From them, 58% remained stable and 42% decline hippocampus volume. No serious adverse events related with neuroEPO were reported.ConclusionNeuroEPO improved clinical outcomes in patients with Alzheimer’s disease with good security profile. Therefore, it could be useful in the treatment of these kind of patients.