Nasal Administration of the NeuroEPO in Parkinson Disease: Short-term Tolerance Physician Lead Trial
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 26
- 试验地点
- 2
- 主要终点
- Number of participants with local nasal events
研究概览
简要总结
Treatment strategies in Parkinson's disease (PD) can improve a patient's quality of life but cannot stop the progression of PD. The investigators are looking for different alternatives that modify the natural course of the disease and recent research has demonstrated the neuroprotective properties of erythropoietin. In Cuba, the Center for Molecular Immunology (CIM) is a cutting edge scientific center where the recombinant form (EPOrh) and recombinant human erythropoietin with low sialic acid (NeuroEPO) are produced.
详细描述
Delivery of therapeutic agents as erythropoietin (EPO) into Central Nervous System through intranasal route could benefit patients with neurological disorders. A new nasal formulation containing a non-hematopoietic recombinant EPO (NeuroEPO) has shown neuroprotective actions in preclinical models and the safety trial of NeuroEPO was evaluated for the first time in humans in Cuba.
The researchers previously conducted a proof of concept clinical trial, administering EPOrh subcutaneously where the neuropsychological performance was measured as a secondary endpoint. This protocol has the register number NCT01010802. Results showed an increased neuropsychological performance of patients after administration as compared to before administration results.
In this protocol two institutions were leading a physician clinical trial.The two institutions: International Center for Neurological Restoration (CIREN from Centro Internacional de Restauracion Neurologica from Spanish) where the recruitment and clinical evaluation of the PD patients will be done and the Center for Molecular Immunology, who was the promoter.
Randomisation will be performed by the CIM to assign the patients to groups. The groups will receive neuroEPO or placebo with identical organoleptic characteristics. The informed consent of all patients will be obtained before the start of the trial after the selection and primary evaluation of the inclusion criteria.
The dose of neuroEPO will be a vial with a dose of 1 mL/1mg administered intra-nasally for five consecutive weeks. The placebo group was administered 1 mL of an intranasal inert solution for the same period of time.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
盲法说明
Randomization will be performed by the promoter institution Centro Inmunologia Molecular from Spanish CIM who assigned the selected patients to two groups neuroEPO and placebo giving a code. The specialists at CIREN didn't have access to the information. Both groups received a vial of the molecule with identical organoleptic characteristics.
入排标准
- 年龄范围
- 40 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient who fulfilled the London Brain Bank's operational criteria for PD
- •Willing to participate in the study;
- •≥1 year since disease onset;
- •Good response to antiparkinsonian treatment with levodopa (>30% change in motor score on the motor section of the Unified Parkinson's Disease
- •no prior poly globulin (hematocrit ≤50%);
排除标准
- •Refusal to participate;
- •Known hypersensitivity to products derived from eukaryotes or hypersensitivity to human albumin;
- •Pregnancy or breastfeeding;
- •Hypertension;
- •Immunosuppressant, androgen or anabolic steroid treatment in the month prior to recruitment;
- •Sepsis or active infection;
- •Active acute or chronic inflammatory diseases;
- •Haematological diseases, such as sickle cell disease, myelodysplastic syndromes, active clotting or bleeding disorders;
- •Malignant tumor or cancer treatment;
- •Alcoholism or drug addiction in the two years prior to inclusion assessment.
- •Significant cognitive decline as measured by clinical assessment, DRS (Dementia Rating Scale) and the Diagnostic and Statistical Manual of Mental Disorders (DSM-IV).
研究组 & 干预措施
NeuroEPO
The dose of NeuroEPO was a vial with a dose of 1 mL/1mg administered intra-nasally for five consecutive weeks.
干预措施: NeuroEPO (Drug)
Placebo
The dose of placebo was a vial with a dose of 1 mL/1mg of an intranasal inert solution administered intra-nasally for five consecutive weeks.
干预措施: NeuroEPO (Drug)
结局指标
主要结局
Number of participants with local nasal events
时间窗: day 1-5
the administration of the drug is intra-nasal for that reason the first adverse event to study is the irritation of the nose. The presence of toxicity signs in the nasal mucous, such as: redness, swelling and nasal congestion was evaluated by means of thorough medical examination of the nasal cavity by the same Otorhinolaryngology Specialist.
Number or participants with increment in the haematological and biochemistry parameters
时间窗: day 0 day 5 and day 14
the blood tests were obtained to evaluate hematological counts (reticulocytes, hemoglobin, hematocrit, leukocytes), coagulation parameters (platelet count, partial thromboplastin and prothrombin times) and blood chemistry (glycemia, creatinine, urea, liver enzymes)
Change from Baseline Systolic Blood Pressure after each intervention
时间窗: day 1-5
The vital signs and the physical checkup was permanent before and after each application. The change of the baseline systolic blood pressure could be a criteria for suspension of the treatment
次要结局
未报告次要终点
研究者
Ivonne Pedroso Ibanez
Head of Movement Disorders Clinic
International Center for Neurological Restoration, Cuba