A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Design, Prospective, Phase IIa Exploratory Clinical Trial to Evaluate the Efficacy and Safety of SC Administration of GV1001 0.56 or 1.12 mg/Day in Patients With PSP
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 78
- 试验地点
- 5
- 主要终点
- Change From the Baseline in the Total Score of PSP-rating Scale
研究概览
简要总结
The study will be conducted by the Sponsor to evaluate the efficacy and safety of GV1001 (0.56 mg and 1.12 mg) administered subcutaneously as a treatment for Progressive Supranuclear Palsy, (PSP). In 75 patients diagnosed with PSPR Richardson(PSP-RS) or PSP-Parkinsonism (PSP-P) at five hospitals in Korea, subcutaneous administration of GV1001 0.56 or 1.12 mg/day will be conducted with multicenter, randomized, double-blind, placebo-controlled, parallel design, prospective phase 2a.
详细描述
This is a 24-week, multicenter, randomized, double-blind, placebo-controlled, parallel design, prospective, Phase 2a exploratory clinical study. If the subject and/or the subject's representative provide a written consent to participate in this clinical study, the required examinations and tests will be performed at the screening visit, and the screening period will run for 4 weeks or shorter. Subjects who are ultimately determined as eligible by the inclusion/exclusion criteria after screening will be randomized at a 1:1:1 ratio to Study Group 1 (GV1001 0.56 mg/day), Study Group 2 (GV1001 1.12 mg/day), or the placebo group depending on the study site in which they are enrolled. Depending on the randomization results, subjects will be administered the investigational product (study drug or placebo) once weekly for the first 4 weeks (1 month), and then administered 10 times at 2-week intervals for 20 weeks (5 months) for a total of 14 doses over 24 weeks (6 months). All subjects will visit the institution according to the planned clinical study schedule to receive the investigational product and to be evaluated for efficacy and safety. To ensure the objectivity and accuracy of the study results, the individuals evaluating efficacy will be limited to neurologists who have been sufficiently educated and trained, and the collection of efficacy and safety evaluation data and biomarkers will be performed in a consistent order at each visit.
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
- 41 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged ≥41 years to ≤ 85 years.
- •Clinical diagnosis of probable progressive supranuclear palsy (PSP).
- •Patient is on a stable therapy for a neurological drug for at least 1 month prior to screening visit.
- •Patients who are able to walk 3 meters or more independently or with assistive devices.
- •Score 15 points ≥ on the Korean Mini-Mental Status Exam (K-MMSE) at the screening visit.
- •Have reliable caregiver to accompany participant to all study visits.
- •Patients and/or their representatives who have voluntarily provided a written consent for participation in this clinical study.
排除标准
- •Patients who have Presence of structural lesions or Suspected concurrent onset of central nervous system diseases based on the CT/MRI scan results and neurological examinations performed within 12 months of screening or at screening.
- •Patients with a history of known or suspected seizures.
- •Patients with a recent unexplained loss of consciousness within 3 months prior to screening or a history of significant head trauma with loss of consciousness.
- •Patients with acute or unstable cardiovascular disease, uncontrolled hypertension, uncontrolled diabetes, or any other medical condition that can interfere with completing the clinical study.
- •Patients with hypersensitivity reactions to the ingredients of the investigational product.
- •Patients with a history of cancer within 5 years prior to screening.
- •Patients with abnormal renal function.
- •Patients with severe liver function abnormalities.
- •Patients weighing ≤35 kg.
- •Among the female subjects who does not agree to use proper contraception.
- •Pregnant or breastfeeding women.
- •Patients who participated in another clinical study within 4 weeks prior to screening and were administered investigational products or were applied investigational medical devices.
- •Patients who were administered the study drug (GV1001) of this clinical study within 12 months prior to screening.
- •Patients who participated in a clinical study for progressive supranuclear palsy within 6 months prior to screening.
- •Other patients judged by the investigator as ineligible to participate in this clinical study.
研究组 & 干预措施
Placebo
Placebo SC injection will be administered once weekly for 4 weeks then every 2 weeks through Week 24 for double blind phase.
干预措施: GV1001 Placebo (Drug)
GV1001 0.56 mg
GV1001 0.56 mg SC injection will be administered once weekly for 4 weeks then every 2 weeks through Week 24 for double blind phase.
干预措施: GV1001 0.56mg (Drug)
GV1001 1.12 mg
GV1001 1.12 mg SC injection administered once weekly for 4 weeks then every 2 weeks through Week 24 for double blind phase.
干预措施: GV1001 1.12mg (Drug)
结局指标
主要结局
Change From the Baseline in the Total Score of PSP-rating Scale
时间窗: 24 weeks(6 months)
Change from the baseline in the total score of PSP-rating scale(Progressive Supranuclear Palsy Rating Scale) after 24 weeks (6 months) of investigational product administration. The possible total scores range from 0 to 100 with a higher score indicating severely impaired cognitive function.
次要结局
- Change From the Baseline in the Total Score of PSP-rating Scale(12 Weeks(3 months))
- Change From the Baseline in the Montreal Cognitive Assessment - Korea (MoCAK)(12 weeks (3 Months))
- Change From the Baseline in the Montreal Cognitive Assessment - Korea (MoCAK)(24 weeks(6 months))
- Change From the Baseline in the Korean Frontal Assessment Battery (K-FAB)(12 weeks(3 months))
- Change From the Baseline in the Korean Frontal Assessment Battery (K-FAB)(24 weeks(6 months))
- Change From the Baseline in the England & Schwab Activity of Daily Living (ES ADL) Scale(12 weeks(3 months))
- Change From the Baseline in the England & Schwab Activity of Daily Living (ES ADL) Scale(24 weeks(6 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (History)(12 weeks(3 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (History)(24 weeks(6 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Mentation)(12 weeks(3 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Mentation)(24 weeks(6 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Bulbar)(12 weeks(3 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Bulbar)(24 weeks(6 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Ocular Motor)(12 weeks(3 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Ocular Motor)(24 weeks(6 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Limb Motor)(12 weeks(3 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Limb Motor)(24 weeks(6 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Midline/Gait)(12 weeks(3 months))
- Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Midline/Gait)(24 weeks(6 months))
- Change From the Baseline in the Score of Each Item of the PSP-rating Scale(Item 12_Dysarthria)(12 weeks(3 months))
- Change From the Baseline in the Score of Each Item of the PSP-rating Scale(Item 12_Dysarthria)(24 weeks(6 months))