A Single Arm, Open-Label Phase 1 Study to Evaluate the Safety and Tolerability of ISC-hpNSC Injected Into the Striatum and Substantia Nigra of Patients With Parkinson's Disease

Cyto Therapeutics Pty Limited1 个研究点分布在 1 个国家目标入组 12 人2016年7月1日

适应症Parkinson Disease

概览

阶段: 1 期
干预措施: 未指定
疾病 / 适应症: Parkinson Disease
发起方: Cyto Therapeutics Pty Limited
入组人数: 12
试验地点: 1
主要终点: Incidence of treatment-emergent adverse events (TEAEs), serious TEAEs, related TEAEs, severe TEAEs
状态: 已完成
最后更新: 3个月前

概览研究者入排标准结局指标研究点相似试验

概览

简要总结

This study will evaluate the safety of an investigational cell transplantation therapy, ISC-hpNSC, in patients with Parkinson's disease. All patients will receive the therapy, which consists of human neural stem cells. Three dose levels will be examined in the study.

详细描述

ISC-hpNSC is a cellular therapeutic consisting of human parthenogenetic neural stem cells (hpNSC). ISC-hpNSC will be injected intracerebrally to the striatum and substantia nigra of patients with Parkinson's disease (PD). The study will enroll 4 patients for cell injection at each of three different doses. A total of 12 patients with moderate to severe PD will be treated. Each patient receives a single dose. The main objective of the study is to evaluate the safety of the cell transplantation.

注册库

clinicaltrials.gov

开始日期

2016年7月1日

结束日期

2025年9月1日

最后更新

3个月前

研究类型

Interventional

研究设计

Single Group

性别

All

研究者

发起方

Cyto Therapeutics Pty Limited

责任方

Sponsor

入排标准

入选标准

•Signed informed consent form (ICF) indicating the patient has been informed of the procedures to be followed, the experimental nature of the therapy, alternatives, potential benefits, side-effects, risks, and discomforts
•Patient diagnosed with idiopathic PD of ≤ 13 years duration, as defined by the United Kingdom (UK) Parkinson's Disease Society Brain Bank criteria
•Outpatients (male and female) 30 - 70 years old. Females must be of non-child bearing potential, or with a negative pregnancy test and not breast-feeding
•Patients receiving a stable dose of levodopa for at least 3 months with the expectation that the treatment will remain unchanged throughout the course of the patient's participation in the trial
•Patients receiving an anti-parkinsonian treatment at a stable dose for at least 3 months with the expectation that the treatment will remain unchanged throughout the course of the patient's participation in the trial
•Hoehn and Yahr stage II-IV during "ON" time
•Unified Parkinson's Disease Rating Scale (UPDRS) motor score (Part III) in the "OFF" state ≤ 49
•Positive dopaminergic response of ≥ 33% decrease in UPDRS motor scores between "OFF" and "ON" states at screening, and unequivocal clinical off periods
•Patient is experiencing motor fluctuations with at least two cumulative hours of daily "OFF" -time during the waking period, which is measured on at least two consecutive days
•History of anti-parkinsonian treatment with sufficient doses of levodopa

排除标准

•Mild cognitive impairment of dementia (MOCA score \< 26)
•The extent or severity of the disease is not measurable
•Severe dyskinesia in the "OFF" or "ON" states (violent dyskinesias, incompatible with any normal motor task)
•Pre-existing medical conditions such as bleeding disorders, septicemia, major cardiovascular, cerebrovascular or psychiatric disease
•Any current or relevant previous history of serious, severe or unstable physical or psychiatric illness or medical disorder that may make the participant unlikely to fully complete the study (depression, anxiety, cognitive impairment or impulse control disorder)
•Clinically significant abnormal hematologic evaluation (blood count, partial thromboplastin time (PTT)), blood chemistry (glucose, blood urea nitrogen (BUN), creatinine, electrolytes), liver function tests (aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma glutamyl transpeptidase (GGTP), total protein, bilirubin)
•Any active infectious disease of any nature, including clinically active viral infections (seropositive for Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B Virus (HBV) and Syphilis
•Severe obesity
•Previous intracranial surgery, including deep-brain stimulation
•History of seizures

结局指标

主要结局

Incidence of treatment-emergent adverse events (TEAEs), serious TEAEs, related TEAEs, severe TEAEs

时间窗: 12 month

次要结局

Change in UPDRS score from baseline(Baseline and 12 months)
Proportion of patients with improvement defined as any reduction in UPDRS motor score(12 months)