Estimates of the Short-term Efficacy of Talineuren (TLN) and Placebo in Patients With Parkinson Disease: A Randomized, Placebo-controlled, Double-blinded, Parallel 2-arm, Multi-centre Pilot Trial
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Movement Disorder Society Unified Parkinson's Disease Rating Scale score
研究概览
简要总结
This study is double-blinded placebo controlled to estimate the short-term efficacy of Talineuren. The investigational Medicinal Product (IMP) is administrated 18 times intravenously as an add-on therapy to the standard of care Parkinson medication.
Talineuren is a liposomal formulation containing GM1 (monosialotetrahexosylganglioside) as the pharmacological active substance.
The results of this pilot study are essential for the sample size calculation of a subsequent larger phase II/III trial.
详细描述
The ganglioside lipid GM1 (monosialotetrahexosylganglioside) has attracted attention in scientific literature as a promising neuroprotective agent. Research suggests that GM1 ganglioside holds promise not only in the treatment of neurodegenerative disorders like Parkinson disease (PD) and Alzheimer's disease but also in promoting nerve regeneration post-injury. Furthermore, investigations into its potential to improve cognitive function and memory underscore its versatility as a therapeutic agent. Numerous clinical studies have demonstrated its therapeutic potential in treating (PD) patients.
Talineuren (TLN) represents a novel approach to harnessing the therapeutic benefits of GM1. TLN is a liposomal formulation, comprising GM1 as its pharmacologically active ingredient, which is expected to cross the blood-brain barrier more efficiently as free GM1 and therefore is able to deliver more GM1 to the brain. This innovative composition is designed to optimize the neuroprotective effects of GM1.
Study Description:
This study is designed as a double-blinded, placebo-controlled trial to evaluate the short-term efficacy of TLN in PD management. The investigational Medicinal Product (IMP), TLN, is weekly intravenously administered 18 times as an add-on therapy alongside patients' current standard-of-care PD medication. Talineuren, encapsulating GM1 within liposomes, is anticipated to facilitate enhanced delivery and bioavailability of the neuroprotective agent, GM1.
Objectives:
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
- 30 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Informed consent as documented by signature.
- •Male and female subjects, aged 30 to 85 years.
- •Confirmed PD according to British brain bank criteria.
- •Hoehn and Yahr Stage 0 - 2.5 on medication.
- •Stable dopaminergic PD treatment (including DBS) for a month at least.
- •Absence of dementia confirmed by cognitive testing (MoCA ≥24).
排除标准
- •Previous treatment with Talineuren (i.e. participants from NEON trial are not allowed)
- •Contraindications to the class of drugs under study, e.g., known hypersensitivity or allergy to class of drugs or the investigational products.
- •Women who are pregnant or breast feeding, or planning to become pregnant during the course of the trial or in the 12 weeks following the trial.
- •Lack of safe contraception, defined as:
- •Female participants of childbearing potential, not willing to use double method of contraception (hormonal and mechanical) for the entire study duration.
- •Note: Female participants who are surgically sterilised / hysterectomised or post-menopausal for longer than 2 years are not considered as being of child bearing potential.
- •Male participants, not using and not willing to use a medically reliable method of contraception for the entire study duration, such as condoms or who are not using any other method considered sufficiently reliable by the investigator in individual cases.
- •Other clinically significant concomitant diseases states (e.g., renal failure, hepatic dysfunction, cardiovascular disease etc.) that is are not under stable control.
- •Known or suspected non-compliance, drug or alcohol abuse.
- •Inability to follow the procedures of the trial, e.g., due to language problems, psychological disorders etc. of the participant.
- •Participation in another trial with an investigational drug within the 30 days preceding and during the present trial.
- •Enrolment of the investigator, his/her family members, employees and other dependent persons.
- •Subject has an atypical parkinsonian syndrome or secondary parkinsonism.
- •Patients with comorbidity that may interfere with the course of the trial.
- •Patients who are not considered to be eligible to participate in clinical trial by the investigator.
- •Patients in adjustment of deep brain stimulation (DBS) parameters
- •Patients with known impaired granulopoiesis
研究组 & 干预措施
Talineuren
Participants receive standard of care PD treatment + 720 mg of Talineuren i.v. weekly for 18 infusions (18 weeks).
干预措施: Talineuren (Drug)
Placebo
Participants receive standard of care PD treatment + placebo (0.9% NaCl) i.v. weekly for 18 infusions (18 weeks).
干预措施: Placebo (Drug)
结局指标
主要结局
Movement Disorder Society Unified Parkinson's Disease Rating Scale score
时间窗: Baseline, week 7, 11, 15, 19 and 22
The Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) will be used by the patient and physician to evaluate various symptoms of Parkinson disease including non-motor and motor experiences of daily living and motor complications. The MDS-UPDRS Scale consists of 4 parts: * Part 1 (Nonmotor aspects of experiences of daily living) with 13 items. * Part 2 (Motor aspects of experiences of daily living) with 13 items. * Part 3 (Motor examination) with 18 items. * Part 4 (Motor complications) with 6 items. Each item is rated with 0=normal, 1=slight, 2=mild, 3=moderate, 4=severe. The lower the score, the fewer / less severe the symptoms.
次要结局
- Montreal Cognitive Assessment score(baseline, week 19)
- Change in Levodopa equivalent daily dose(Through study completion, an average of 22 weeks)
- Parkinson disease Quality of Life Questionnaire score(Baseline, week 19)