NCT05830396招募中2 期
GRoningen Early-PD Ambroxol Treatment (GREAT) Trial: A Randomised, Double-blind, Placebo-controlled, Singlecenter Trial With Ambroxol in Parkinson Patients With a GBA Mutation
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 80
- 试验地点
- 1
- 主要终点
- MDS-UPDRS3 motor scale
研究概览
简要总结
The most common genetic risk factor for Parkinson's Disease is a heterozygous mutation of the GBA1 gene, encoding the lysosomal enzyme glucocerebrosidase (GCase). Reduced GCase activity is associated with aggregation of the protein alpha synucleine (aSyn) in the central nervous system, which is related to the pathological cause of PD. Ambroxol is a mucolytic expectorant that appears to facilitate the refolding of the misfolded GBA protein thats acts as a chaperone for GCase.
This randomized placebo-controlled trial aims to investigate the disease-modifying properties of ambroxol in PD patients with a GBA1-mutation. Patients will undergo motor and cognitive tests, as well as imaging and blood tests.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Parkinson's disease, according to Movement Disorders Society (MDS) criteria (27)
- •Disease duration of 10 years or less at time of inclusion
- •PD patients carrying a GBA1 mutation
- •Able to write written informed consent, understanding study protocol and perform protocol related actions
- •Willing and able to self-administer oral ambroxol or placebo medication
排除标准
- •The refusal to be informed about an unforeseen clinical finding
- •Use of an implanted Deep Brain Stimulation (DBS) system
- •Confirmed dysphagia that would preclude self-administration of ambroxol or placebo tablets
- •History of known sensitivity to the study medication
- •Pregnant or breastfeeding women
- •Participants of childbearing potential that would not use adequate birth control, consisting of a negative pregnancy test at the screening visit and use of accepted contraceptive methods defined as highly effective while participating in the study
- •MRI incompatible implants in the body
- •Any clinically significant or unstable medical or surgical condition that in the opinion of the principal investigator may put the participant at risk when participating in the study or may influence the results of the study or affect the participant's ability to take part in the study, as determined by medical history, physical examinations, electrocardiogram (ECG), or laboratory tests. Such conditions may include:
- •Impaired renal function (a positive urine dipstick test, and laboratory values below or above: a eGFR <45 ml/min 1,73M2, Sodium 135-145 mmol/L, Potassium 3.5-5.0 mmol/L, Urea 2.5-7.5mmol/L).
- •Moderate/severe hepatic impairment (laboratory values below or above: ASAT 0- 80U/L, ALAT0-90 U/L, GGT > 80 U/L, Alkaline Phosphatase 35-210 U/L).
研究组 & 干预措施
Ambroxol
Experimental
Ambroxol 1800mg/day
干预措施: Ambroxol Hydrochloride (Drug)
Placebo
Placebo Comparator
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
MDS-UPDRS3 motor scale
时间窗: 60 weeks
Motor scale developed for PD patients, 0-132. 0 means good performance, 132 means very bad performance
次要结局
- Safety and tolerability measured by incidence of adverse events and possible side effects(all throughout the study. specifically at: 1, 2, 3, 12, 24, 36, 48, 60 weeks)
- Glucocerebrosidase (GCase) activity in blood mononuclear cells(0, 12, 60 weeks)
- Striatal F-DOPA uptake as measured by [18] F-DOPA PET scan(0, 60 weeks)
- Cognition, using the Montreal Cognitive Assessment (MoCA)(0, 60 weeks)
- fMRI resting state to investigate the functional architecture and structural MRI for PET-scan(0, 60 weeks)
- Quality of Life (PDQ-39 questionnaire)(0, 60 weeks)
- Non Motor Symptoms (NMSS scale)(0, 60 weeks)
研究者
研究点 (1)
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