Multicenter, Randomized, Double-Blind, Sham Surgery-Controlled Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) to Assess the Efficacy and Safety of Bilateral Intraputaminal (IPu) Delivery in Subjects With Idiopathic Parkinson's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 58
- 试验地点
- 9
- 主要终点
- UPDRS Part III OFF
研究概览
简要总结
The purpose of this double blind study is to determine whether CERE-120 (adeno-associated virus serotype 2 [AAV2]-neurturin [NTN]) is effective and safe in the treatment of patients with idiopathic Parkinson's Disease. CERE-120 is administered via bilateral stereotactic injections targeting the putaminal region of the brain. The design of this study involves approximately 34 patients receiving CERE-120 treatment via stereotactic surgery and approximately 17 patients receiving sham stereotactic surgery (no CERE-120 administered).
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
- 35 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of bilateral, idiopathic Parkinson's Disease (PD) based on UK Brain Bank criteria with motor complications despite adequate oral antiparkinsonian therapy.
- •At least 5 years disease duration, relative to the anticipated date of surgery, since diagnosis of PD.
- •Males or nonpregnant females 35-75 years of age, inclusive.
- •A UPDRS motor scale score of 30 or greater in the practically defined off condition during the 30-day eligibility evaluation period.
- •Stable doses of antiparkinsonian medications and parkinsonian features for the 60-day period preceding the surgical procedure.
- •No conditions that would render the subject unsuitable for surgery, or that would interfere with any of the assessments of efficacy or safety in this trial.
- •Subject's informed consent prior to the performance of any study-specific procedures.
排除标准
- •Subjects with atypical or secondary parkinsonism.
- •Any subject, in the judgment of the investigator, for whom participation in the study would pose a safety risk including, but not limited to, a history of any clinically significant medical, psychiatric, or laboratory abnormality.
- •History of treatment of PD by any procedure involving intracranial surgery or implantation of a device.
- •MRI of the brain within 12 months before the surgical procedure that indicates the presence of an abnormality that may interfere with the assessments of safety or efficacy or would, in the judgment of the investigator, represent a surgical risk to the subject.
- •Any disorder that precludes a surgical procedure (e.g., signs of sepsis or inadequately treated infection) or alters wound healing.
- •Receipt of antiplatelet agents for at least 10 days prior to the surgical procedure.
- •A score of less than or equal to 27 on the Folstein Mini-Mental examination performed during the eligibility evaluation period or clinical evidence of cognitive impairment that would affect the subject's ability to sign the informed consent or perform any of the protocol required assessments.
- •Chemotherapy, cytotoxic therapy, or immunotherapy within 6 weeks prior to the surgical procedure.
- •Vaccinations within 30 days prior to the surgical procedure.
- •History, within 2 years before the surgical procedure, of drug or alcohol abuse.
- •Treatment with neuroleptics within 1 year before the surgical procedure.
- •Any medical disability (e.g., severe degenerative arthritis, compromised nutritional state, peripheral neuropathy) that would interfere with the assessment of efficacy and safety in this trial or would compromise the ability of the subject to undergo study procedures (e.g., MRI, PET), or give informed consent.
- •History of prior gene transfer therapy.
- •Treatment with an investigational agent within 60 days before the surgical procedure.
研究组 & 干预措施
1
Intracerebral administration of CERE-120
干预措施: CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) (Drug)
2
Sham Neurosurgery
干预措施: Sham Surgery (Procedure)
结局指标
主要结局
UPDRS Part III OFF
时间窗: Change from Baseline to 12 Month Visit
The UPDRS (Unified Parkinson's Disease Rating Scale) is a clinical rating scale that assesses the symptomatic burden of Parkinson's Disease. The scale has four main sections, and each item is scored from a 0 to a 4 (higher number is more severe manifestation). Part III is a subsection devoted to motor function, has 14 questions, resulting in a score range of 0 (unaffected) to 56 (severely affected). The scale is administered by a trained clinician, and patients were assessed in a practically defined "off" condition, 12 hours or more after the last administration of medication.
次要结局
未报告次要终点