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

• ClinicalTrials.gov Identifier: NCT00252850
• Recruitment Status: Completed
• First Posted: November 15, 2005
• Last Update Posted: September 7, 2016
• Sponsor: Ceregene
• Information provided by: Sangamo Therapeutics

Study Description

Brief Summary:

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 Design

• Study Type: Interventional (Clinical Trial)
• Enrollment: 12 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (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
• Study Start Date: June 2005
• Actual Primary Completion Date: March 2007
• Actual Study Completion Date: March 2007

Arms and Interventions

Outcome Measures

Eligibility Criteria

• Ages Eligible for Study: 35 Years to 75 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• 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

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 of Results:

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. doi: 10.1016/S1474-4422(08)70065-6. Epub 2008 Apr 2.

• Responsible Party: Joao Siffert, Chief Medical Officer, Ceregene
• ClinicalTrials.gov Identifier: NCT00252850
• Other Study ID Numbers: CERE-120-01
• First Posted: November 15, 2005
• Last Update Posted: September 7, 2016
• Last Verified: September 2016

Keywords provided by Sangamo Therapeutics:

Parkinson's Disease; Gene Transfer

Additional relevant MeSH terms:

Parkinson Disease; Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Neurodegenerative Diseases