Safety Evaluation of Intravenous Talineuren (TLN) in Parkinson's Disease-affected Patients
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 22
- 试验地点
- 2
- 主要终点
- Occurence of adverse events (safety)
研究概览
简要总结
This study is an open-label, single ascending dose escalation followed by a multiple administration dose at the maximal suitable dose (MSD). The investigational Medicinal Product (IMP) is given as an add-on therapy.
Talineuren consists of GM1 (monosialotetrahexosylganglioside), the pharmacologically active ingredient, associated with a proprietary lipid formulation assembled as liposomes.
The primary objective is to demonstrate the safety of TLN administration intravenously in Parkinson patients.
Secondary objectives are the determination of the maximal suitable dose based on the safety profile and preliminary efficacy, as well as the determination of the pharmacokinetics (PK) profile.
详细描述
The ganglioside lipid GM1 has been described in the literature as a neuroprotective agent.
Several clinical studies have shown that GM1 improves the condition of Parkinson's disease patients.
Talineuren consists of the pharmacologically active ingredient GM1, associated with a proprietary lipid formulation assembled as liposomes. Talineuren has been developed to improve the delivery and bioavailability of GM1.
The primary objective of this trial is to demonstrate the feasibility and safety of intravenous Talineuren administration in Parkinson's disease patients.
The secondary objectives are:
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
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.
- •Confirmed Parkinson's disease according to British brain bank criteria.
- •Hoehn and Yahr Stage 0 - 2.5 on medication.
- •Stable on PD treatment for a month at least.
- •Absence of dementia confirmed by cognitive testing (MoCA >25).
排除标准
- •Contraindications to the class of drugs under study, e.g., known hypersensitivity or allergy to class of drugs or the investigational product.
- •Women who are pregnant or breast feeding, or planning to become pregnant during the course of the trial or in the 3 months following the trial.
- •Lack of safe contraception in women with childbearing potential
- •Other clinically significant concomitant disease states (e.g., renal failure, hepatic dysfunction, cardiovascular disease, etc) that is not under stable control.
- •Subject has an atypical parkinsonian syndrome or secondary parkinsonism.
- •Patients with comorbidity that may interfere with the course of the trial.
研究组 & 干预措施
Talineuren dose escalation
14 doses of GM1 Ganglioside 6, 12, 60, 120, 180, 240, 300, 360, 420, 480, 540, 600, 660, 720 mg. Optional treatment prolongations for 16 weeks (Amendment 2), 8 months (Amendment 3), 4 months (Amendment 4) and 12 months (Amendment 5).
干预措施: Talineuren (Drug)
Talineuren dose consolidation with intrapatient dosing (additional patients)
8 months repeated doses of 720mg GM1 Ganglioside (Amendment 3).
干预措施: Talineuren (Drug)
Talineuren repeated dose
8 repeated doses of GM1 Ganglioside tbd from the escalation dose (maximum suitable dose). Optional treatment prolongations for 16 weeks (Amendment 2), 8 months (Amendment 3), 4 months (Amendment 4) and 12 months (Amendment 5).
干预措施: Talineuren (Drug)
结局指标
主要结局
Occurence of adverse events (safety)
时间窗: 8 to 134 weeks
Number and kinds of adverse events (AEs)
Occurence of serious adverse events (safety)
时间窗: 8 to 134 weeks
Number and kinds of serious adverse events (SAEs)
Occurence of other safety-related signs (safety)
时间窗: 8 to 134 weeks
Number and kinds of other safety-related signs
次要结局
- Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)(8 to 134 weeks)
- Time of Maximum Drug Concentration (Tmax) in serum(8 to 15 weeks)
- half-life (t1/2)(8 to 15 weeks)
- Volume of distribution (Vd)(8 to 15 weeks)
- Levodopa challenge (LDC) test(8 to 134 weeks)
- Change in Parkinson's medication(8 to 134 weeks)
- Starkstein Apathy Scale (SAS)(8 to 134 weeks)
- Non-Motor Symptoms Questionnaire (NMSQuest)(8 to 134 weeks)
- Epworth Sleepiness Scale (ESS)(8 to 134 weeks)
- Area Under the Curve to infinity (AUCinf.) in serum(8 to 15 weeks)
- Clearance (CL)(8 to 15 weeks)
- Parkinson's Disease Questionnaire (PDQ-39)(8 to 134 weeks)
- Montreal Cognitive Assessment (MoCA)(8 to 134 weeks)
- Beck's Depression Inventory (BDI)(8 to 134 weeks)
- Maximum Observed Drug Concentration (Cmax) in serum(8 to 15 weeks)