Safety of CERE-120 (AAV2-NTN) in Subjects With Idiopathic Parkinson's Disease
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00252850|
Recruitment Status : Completed
First Posted : November 15, 2005
Last Update Posted : September 7, 2016
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.
|Condition or disease||Intervention/treatment||Phase|
|Parkinson's Disease||Genetic: CERE-120: AAV2-NTN||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Study Start Date :||June 2005|
|Actual Primary Completion Date :||March 2007|
|Actual Study Completion Date :||March 2007|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00252850
|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|