A Phase 1/2 Trial Assessing the Safety and Efficacy of Bilateral Intraputaminal and Intranigral Administration of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) in Subjects With Idiopathic Parkinson's Disease
The purpose of this study is to evaluate the safety and potential benefits of CERE-120 in the treatment of Parkinson's disease. CERE-120 is an experimental drug that consists of an adeno-associated virus (AAV) that was engineered to carry the human gene for neurturin, a neurotrophic (growth) factor. Similar to other growth factors (such as GDNF), neurturin is capable of restoring function and protecting brain cells from further damage. The virus used in CERE-120 is not known to cause disease in people. CERE-120 is delivered directly to the brain cells most affected in Parkinson's disease - the dopamine producing neurons. CERE-120 is injected during brain surgery. Once in place, CERE-120 continuously produces neurturin.
Stanford is not currently accepting new patients for this trial. You may want to check clinicaltrials.gov to see if other locations are recruiting.
- biological : CERE-120: Adeno-Associated Virus Delivery of Neurturin
- procedure : Sham Surgery
Phase: Phase 1/Phase 2
Ages Eligible For Study:
- 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 ?-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