NCT07498426招募中3 期
A Phase III, Randomized, Placebo-controlled, Parallel Group, Double-blind Study to Evaluate the Efficacy and Safety of NIO752 in Participants With Progressive Supranuclear Palsy Followed by an Open Label Extension
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 300
- 试验地点
- 42
- 主要终点
- Change from baseline in the mPSPRS-10 score
研究概览
简要总结
This Phase III study is intended to evaluate the efficacy and safety of NIO752 in participants with Progressive Supranuclear Palsy (PSP). Eligible participants will be randomized to receive either NIO752 or placebo followed by an open-label extension.
详细描述
This is a randomized, double-blind, placebo-controlled, parallel group, study to evaluate the efficacy of NIO752 in participants with Progressive Supranuclear Palsy followed by an open-label extension (OLE).
The participants with or without symptomatic therapy will be randomly allocated to either NIO752 or placebo treatment in a 2:1 randomization ratio.
Upon completion of the core double-blind treatment period, participants will be offered to continue with NIO752 treatment in the OLE.
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 81 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent must be obtained prior to participation in the study.
- •Male or female participants, age between 41-81 yrs inclusive.
- •Diagnosis of mild-moderate, probable/possible PSP Richardson syndrome as per MDS-PSP 2017 criteria with symptoms onset < 5 years.
- •PSPRS total score less than 40 at Baseline.
- •Reliable study partner such as spouse, sibling, close friend, or caregiver able and willing to provide accurate information (including clinical symptoms and medical history) about the participant and to participate in study visits and informant-based assessments for the duration of the study. A reliable study partner is expected to spend enough time (at least 5 hours per week) with the study participant.
- •Participant is able to ambulate defined as the ability to take at least 10 steps independently or with minimal assistance (stabilization of one arm to minimize fall risk).
- •Mini Mental State Examination (MMSE) score ≥ 20 at Screening.
排除标准
- •Diagnosis of other significant neurological or psychiatric disorders including (but not limited to) Parkinsons' Disease (which has not subsequently been revised to a diagnosis of PSP); Alzheimer's disease (AD), dementia with Lewy bodies; prion disease; any psychotic disorders; severe Major depressive disorder; seizure; brain tumor or other space-occupying lesion; history of clinically significant stroke (e.g., stroke with permanent neurological deficit); history of head injury with loss of consciousness for at least 15 minutes within the past 20 years.
- •Diagnosis of amyotrophic lateral sclerosis or other motor neuron diseases.
- •Diagnosis of cerebellar ataxia, choreoathetosis, and early symptomatic autonomic dysfunction.
- •History of or screening brain MRI scan indicative of significant abnormality, including, but not limited to, prior hemorrhage or infarct >1 cm3, >3 lacunar infarcts, cerebral contusion, aneurysm, vascular malformation >1 cm3, subdural hematoma, hydrocephalus, and space-occupying lesion (e.g., abscess or brain tumor).
- •Contraindications to undergo MRI procedure, including metal (ferromagnetic) implants and/or a cardiac pacemaker that is not compatible with MRI.
- •Medical conditions that would, as per Investigator's judgement, prevent the participant from undergoing lumbar puncture, including but not limited to:
- •Known allergy to local anesthetic
- •History of back surgery (with the exception of microdiscectomy or laminectomy over 1 level)
- •Spinal deformities
- •Current dermatological infection at the lumbar puncture spot and/or significant skin alterations at the planned puncture place
- •Risk of increased or uncontrolled bleeding and/or risk of bleeding that if not managed optimally, could place a participant at an increased risk for procedural bleeding. These could include, but are not limited, to anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms) and underlying disorders of coagulation, platelet function or platelet count (e.g. abnormal coagulation parameters, hemophilia, Von Willebrand's disease, liver disease).
- •History of deep brain stimulator surgery other than sham surgery for participation in a deep brain stimulation clinical trial.
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
Placebo
Placebo Comparator
Placebo in solution
干预措施: Placebo (Drug)
NIO752
Experimental
NIO752 solution
干预措施: NIO752 (Other)
结局指标
主要结局
Change from baseline in the mPSPRS-10 score
时间窗: Baseline, Week 72
The 10-item Progressive Supranuclear Palsy Rating Scale (mPSPRS-10) is a modified version of the original 28 item PSPRS developed to improve clinical meaningfulness and statistical performance. The 10 items measure three key motor domains: gait, limb function, and bulbar. The mPSPRS-10 ranges between 0 and 30, with higher scores indicating greater disability.
次要结局
- Change from baseline in the PSPRS-28 items score(Baseline, Week 72)
- Changes from baseline in activities of daily living on the Cortical Basal ganglia Functional Scale (CBFS)(From baseline up to week 72)
- Change from baseline on the PSP-ShoQoL(From baseline up to week 72)
- Ratio to baseline of CSF phospho-Tau-181 and CSF Total-Tau(From baseline up to week 72)
- Change from baseline in Category (or Semantic) Fluency test over time(From baseline up to week 72)
- Change from baseline in Letter (or Phonemic) Fluency test over time(From baseline up to week 72)
- Change from baseline in Symbol Digit Modality Test (SDMT) over time(From baseline up to week 72)
- Change from baseline in Letter-Number Sequencing task (LNS) over time(From baseline up to week 72)
- Ratio to baseline of NfL (CSF)(From baseline up to week 72)
- Changes from baseline in volumes of brain structures as measured by MRI(From baseline up to week 72)
- Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)(From first treatment administration up to 72 weeks)
研究者
研究点 (42)
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