Safety of CERE-120 (AAV2-NTN) in Subjects With Idiopathic Parkinson's Disease - Full Text View

Sponsor:	Ceregene
Information provided by:	Ceregene
ClinicalTrials.gov Identifier:	NCT00252850

Purpose

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	Intervention	Phase
Parkinson's Disease	Genetic: CERE-120: AAV2-NTN	Phase I

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study
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

Resource links provided by NLM:

Genetics Home Reference related topics: familial paroxysmal nonkinesigenic dyskinesia Parkinson disease Help Me Understand Genetics

MedlinePlus related topics: Parkinson's Disease

U.S. FDA Resources

Further study details as provided by Ceregene:

Estimated Enrollment:	12
Study Start Date:	June 2005
Study Completion Date:	March 2007
Primary Completion Date:	March 2007 (Final data collection date for primary outcome measure)

Eligibility

Ages Eligible for Study:	35 Years to 75 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Diagnosis of bilateral idiopathic Parkinson's Disease of at least 5 years duration since diagnosis with motor fluctuations, despite adequate oral antiparkinsonian therapy.
Diagnosis of moderate to severe Parkinson's Disease based on clinical rating scales.
Males or nonpregnant females 35-75 years of age, inclusive.
Stable medication requirements, and clear response to antiparkinsonian medications during the 60-day eligibility evaluation period.
No conditions that would render the subject unsuitable for surgery, or that would interfere with any of the assessments of safety or efficacy in this trial.
Subject's informed consent prior to the performance of any study-specific procedure.

Exclusion Criteria:

A history of any clinically significant medical, psychiatric, or laboratory abnormality for which participation in the study would pose a safety risk to the subject.
History of treatment of Parkinson's disease by any procedure involving intracranial surgery or implantation of a device.
MRI of the brain within 12 months before the anticipated dosing 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, present a surgical risk to the subject.
Any disorder that precludes a surgical procedure or alters wound healing.
A score of less than or equal to 25 on the Folstein Mini-Mental examination performed during the eligibility evaluation period.
Chemotherapy, cytotoxic therapy, or immunotherapy within 6 weeks prior to CERE-120 administration.
Vaccinations within 30 days prior to CERE-120 administration.
History, within two years before the anticipated dosing procedure, of drug or alcohol abuse.
Treatment with nonantiparkinsonian agents that may affect symptoms of Parkinson's disease within 60 days before the anticipated dosing procedure.
Any medical disability that would interfere with the assessment of safety and efficacy in this trial or would compromise the ability of the subject to undergo study procedures (e.g. MRI, PET) or to give informed consent.
History of prior gene transfer therapy.
Treatment with an investigational agent within 60 days before the anticipated dosing procedure.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00252850

Locations

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

Sponsors and Collaborators

Ceregene

Investigators

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

More Information

Publications:

Marks WJ Jr, Ostrem JL, Verhagen L, Starr PA, Larson PS, Bakay RA, Taylor R, Cahn-Weiner DA, Stoessl AJ, Olanow CW, Bartus RT. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol. 2008 May;7(5):400-8. Epub 2008 Apr 2.

Responsible Party:	Ceregene ( Joao Siffert, Chief Medical Officer )
Study ID Numbers:	CERE-120-01
Study First Received:	November 11, 2005
Last Updated:	March 25, 2009
ClinicalTrials.gov Identifier:	NCT00252850 History of Changes
Health Authority:	United States: Food and Drug Administration

Keywords provided by Ceregene:

Parkinson's Disease
Gene Transfer

Additional relevant MeSH terms:

Movement Disorders
Parkinson Disease
Nervous System Diseases
Basal Ganglia Diseases

Central Nervous System Diseases
Parkinsonian Disorders
Neurodegenerative Diseases
Brain Diseases

ClinicalTrials.gov processed this record on February 04, 2010