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

Parkinson's Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00252850
• Other study ID #: CERE-120-01
• Status: Completed
• Phase: Phase 1
• Start date: June 2005
• Est. completion date: March 2007

Study information

• Verified date: September 2016
• Source: Sangamo Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: March 2007
• Est. primary completion date: March 2007
• Accepts healthy volunteers: No
• Gender: All
• Age group: 35 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Parkinson Disease
• Parkinson's Disease

Intervention

Genetic:
• CERE-120: AAV2-NTN

Locations

Country	Name	City	State
United States	Rush University Medical Center	Chicago	Illinois
United States	University of California, San Francisco	San Francisco	California

Sponsors (2)

Lead Sponsor	Collaborator
Sangamo Therapeutics	Ceregene

Country where clinical trial is conducted

United States, 

References & Publications (1)

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 id — View Citation