Double-Blind, Multicenter, Sham Surgery Controlled Study of CERE-120 in Subjects With Idiopathic Parkinson's Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Ceregene
ClinicalTrials.gov Identifier:
NCT00400634
First received: November 15, 2006
Last updated: July 6, 2012
Last verified: July 2012
  Purpose

The purpose of this double blind study is to determine whether CERE-120 (adeno-associated virus serotype 2 [AAV2]-neurturin [NTN]) is effective and safe in the treatment of patients with idiopathic Parkinson's Disease. CERE-120 is administered via bilateral stereotactic injections targeting the putaminal region of the brain. The design of this study involves approximately 34 patients receiving CERE-120 treatment via stereotactic surgery and approximately 17 patients receiving sham stereotactic surgery (no CERE-120 administered).


Condition Intervention Phase
Idiopathic Parkinson's Disease
Genetic: CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])
Procedure: Sham Surgery
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Multicenter, Randomized, Double-Blind, Sham Surgery-Controlled Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) to Assess the Efficacy and Safety of Bilateral Intraputaminal (IPu) Delivery in Subjects With Idiopathic Parkinson's Disease

Resource links provided by NLM:


Further study details as provided by Ceregene:

Primary Outcome Measures:
  • UPDRS Part III OFF [ Time Frame: Change from Baseline to 12 Month Visit ] [ Designated as safety issue: No ]
    The UPDRS (Unified Parkinson's Disease Rating Scale) is a clinical rating scale that assesses the symptomatic burden of Parkinson's Disease. The scale has four main sections, and each item is scored from a 0 to a 4 (higher number is more severe manifestation). Part III is a subsection devoted to motor function, has 14 questions, resulting in a score range of 0 (unaffected) to 56 (severely affected). The scale is administered by a trained clinician, and patients were assessed in a practically defined "off" condition, 12 hours or more after the last administration of medication.


Enrollment: 58
Study Start Date: November 2006
Study Completion Date: November 2008
Primary Completion Date: November 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Intracerebral administration of CERE-120
Genetic: CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])
CERE-120 5.4 x 10^11 vg
Sham Comparator: 2
Sham Neurosurgery
Procedure: Sham Surgery
Bilateral partial thickness burr holes placed, no intraparenchymal injections

  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 (PD) based on UK Brain Bank criteria with motor complications despite adequate oral antiparkinsonian therapy.
  • At least 5 years disease duration, relative to the anticipated date of surgery, since diagnosis of PD.
  • Males or nonpregnant females 35-75 years of age, inclusive.
  • A UPDRS motor scale score of 30 or greater in the practically defined off condition during the 30-day eligibility evaluation period.
  • Stable doses of antiparkinsonian medications and parkinsonian features for the 60-day period preceding the surgical procedure.
  • No conditions that would render the subject unsuitable for surgery, or that would interfere with any of the assessments of efficacy or safety in this trial.
  • Subject's informed consent prior to the performance of any study-specific procedures.

Exclusion Criteria:

  • Subjects with atypical or secondary parkinsonism.
  • Any subject, in the judgment of the investigator, for whom participation in the study would pose a safety risk including, but not limited to, a history of any clinically significant medical, psychiatric, or laboratory abnormality.
  • History of treatment of PD by any procedure involving intracranial surgery or implantation of a device.
  • MRI of the brain within 12 months before the surgical 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, represent a surgical risk to the subject.
  • Any disorder that precludes a surgical procedure (e.g., signs of sepsis or inadequately treated infection) or alters wound healing.
  • Receipt of antiplatelet agents for at least 10 days prior to the surgical procedure.
  • A score of less than or equal to 27 on the Folstein Mini-Mental examination performed during the eligibility evaluation period or clinical evidence of cognitive impairment that would affect the subject's ability to sign the informed consent or perform any of the protocol required assessments.
  • Chemotherapy, cytotoxic therapy, or immunotherapy within 6 weeks prior to the surgical procedure.
  • Vaccinations within 30 days prior to the surgical procedure.
  • History, within 2 years before the surgical procedure, of drug or alcohol abuse.
  • Treatment with neuroleptics within 1 year before the surgical procedure.
  • Any medical disability (e.g., severe degenerative arthritis, compromised nutritional state, peripheral neuropathy) that would interfere with the assessment of efficacy and safety in this trial or would compromise the ability of the subject to undergo study procedures (e.g., MRI, PET), or give informed consent.
  • History of prior gene transfer therapy.
  • Treatment with an investigational agent within 60 days before the surgical 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: NCT00400634

Locations
United States, Alabama
University of Alabama, Birmingham
Birmingham, Alabama, United States, 35233
United States, California
University of California, San Francisco
San Francisco, California, United States, 94143-0138
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
United States, New York
Mount Sinai School of Medicine
New York, New York, United States, 10029
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27705
United States, Ohio
Cleveland Clinic
Cleveland, Ohio, United States, 44195
United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Pennsylvania Hospital
Philadelphia, Pennsylvania, United States, 19107
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Sponsors and Collaborators
Ceregene
Investigators
Study Director: Joao Siffert, M.D. Ceregene
  More Information

Publications:
Responsible Party: Ceregene
ClinicalTrials.gov Identifier: NCT00400634     History of Changes
Other Study ID Numbers: CERE-120-02
Study First Received: November 15, 2006
Results First Received: May 24, 2012
Last Updated: July 6, 2012
Health Authority: United States: Food and Drug Administration

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 August 28, 2014