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

This study has been completed.
Sponsor:
Information provided by:
Ceregene
ClinicalTrials.gov Identifier:
NCT00252850
First received: November 11, 2005
Last updated: March 25, 2009
Last verified: March 2009
  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 1

Study Type: Interventional
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

Resource links provided by NLM:


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

Keywords provided by Ceregene:
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

ClinicalTrials.gov processed this record on July 29, 2014