Safety of CERE-120 (AAV2-NTN) in Subjects With Idiopathic Parkinson's Disease
This is a Phase I dose escalating open-label study designed to assess the safety, tolerability and biologic activity of an in vivo AAV2 mediated delivery of the gene encoding NTN (CERE-120).
Twelve (up to 18) subjects will receive one of two open-label doses of CERE-120 via bilateral stereotactic injections targeting the putaminal region of the brain. Subjects will be enrolled in one of two cohorts, a low-dose cohort of six subjects followed by a high dose cohort of six subjects.
The design of this study is such that the primary objective, the evaluation of safety and tolerability, will be assessed by frequent observations for adverse events, clinical laboratory test results, imaging (MRI), neuropsychometric testing, and evaluations of disease status.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase I, Open-Label Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin[NTN] to Assess the Safety and Tolerability of Intrastriatal Delivery to Subjects With Idiopathic Parkinson's Disease|
|United States, California|
|University of California, San Francisco|
|San Francisco, California, United States, 94143|
|United States, Illinois|
|Rush University Medical Center|
|Chicago, Illinois, United States, 60612|
|Principal Investigator:||William J Marks, Jr., M.D.||University of California, San Francisco|
|Principal Investigator:||Leo Verhagen Metman, M.D., Ph.D.||Rush University Medical Center|