Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease

This is an interventional treatment trial for Idiopathic Parkinson's Disease focused on measuring Parkinson's disease, Movement Disorders, Gene Therapy, Neurotrophic Factors, GDNF, Neurturin, DBS, Dopamine Read More

Inclusion Criteria:

• Males and females, ages 35 to 70 years old (inclusive)
• A diagnosis of idiopathic Parkinson's disease based on UK Brain Bank criteria, including bradykinesia and at least 1 of the following PD features: resting tremor or rigidity
• A Hoehn and Yahr score of no greater than 3 in the "off" condition at Screening
• A robust response to dopaminergic therapy as judged by the investigator based on the UPDRS Part III: Motor Examination
• Experiencing motor complications despite adequate antiparkinsonian therapy
• A stable, optimized regimen of antiparkinsonian medications and stable parkinsonian features for at least 6 weeks prior to Screening
• Subject is willing not to undergo DBS for at least 12 months after the study surgical procedure (Phase 1 subjects) or while the study is blinded (Phase 2 subjects) and the investigator believes that this is medically acceptable
• Medically fit to undergo the study surgical procedure as determined by medical history, clinical and laboratory evaluations, and any other pre-surgical evaluations that are standard at the institution where the subject will undergo surgery
• Physically and mentally capable of performing all protocol-specified assessments and complying with the study visit schedule
• Subjects must be able to travel to study visits alone or able to identify a partner or caregiver who agrees to accompany the subject to the study visits
• Females of childbearing potential must have a negative β-HCG pregnancy test at Screening and again before surgery on Day 0
• All subjects, both male and female, must agree to practice adequate barrier method contraception for at least 6 months after the surgical procedure
• Provides written informed consent to participate before any study-specific procedures are conducted

Exclusion Criteria:

• Atypical or secondary parkinsonism, including, but not limited to, multiple system atrophy (MSA) or progressive supranuclear palsy
• Any subject for whom participation in the study would pose a substantial safety risk
• Any condition that would compromise the ability of the subject to undergo study procedures, including allergy to gadolinium
• Presence of any known brain abnormality that could interfere with the assessment of safety or efficacy or represents a surgical risk to the subject
• Evidence of significant brain atrophy on the Baseline MRI
• History of any cancer other than basal or squamous cell skin cancer within the 3 years prior to Screening
• Any chemotherapy, cytotoxic therapy, or immunotherapy (e.g., IL-2, IL-12, interferon) within the 3 months prior to Screening
• Any prior treatment for PD with a procedure involving intracranial surgery or implantation of a device (e.g. DBS, pallidotomy)
• Any prior treatment for a neurological or psychiatric disorders with a procedure involving the implantation of a device (e.g. spinal cord stimulator, vagus nerve stimulator)
• History of any prior gene transfer therapy
• Treatment with any investigational agent within the 3 months prior to Screening
• Anticipated need for antiplatelet agents or anticoagulation therapy, including gingko biloba, during the 10 days prior to the projected surgery date
• Any vaccinations within the 30 days prior to the projected surgery date Note: Vaccinations are not allowed for 30 days after the surgical procedure, unless deemed necessary by the investigator for the subject's well-being
• Not likely to be available for the duration of the trial, likely to be noncompliant with the protocol, or who are deemed unsuitable by the investigator for any other reason
• Participation in a previous surgical treatment study for Parkinson's disease