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

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ClinicalTrials.gov Identifier: NCT00400634
Recruitment Status : Completed
First Posted : November 17, 2006
Results First Posted : August 14, 2012
Last Update Posted : August 14, 2012
Sponsor:
Information provided by (Responsible Party):
Ceregene

Tracking Information
First Submitted Date  ICMJE November 15, 2006
First Posted Date  ICMJE November 17, 2006
Results First Submitted Date  ICMJE May 24, 2012
Results First Posted Date  ICMJE August 14, 2012
Last Update Posted Date August 14, 2012
Study Start Date  ICMJE November 2006
Actual Primary Completion Date November 2008   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 6, 2012)
UPDRS Part III OFF [ Time Frame: Change from Baseline to 12 Month Visit ]
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.
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00400634 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Double-Blind, Multicenter, Sham Surgery Controlled Study of CERE-120 in Subjects With Idiopathic Parkinson's Disease
Official Title  ICMJE 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
Brief Summary 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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Idiopathic Parkinson's Disease
Intervention  ICMJE
  • Genetic: CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])
    CERE-120 5.4 x 10^11 vg
  • Procedure: Sham Surgery
    Bilateral partial thickness burr holes placed, no intraparenchymal injections
Study Arms  ICMJE
  • Experimental: 1
    Intracerebral administration of CERE-120
    Intervention: Genetic: CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])
  • Sham Comparator: 2
    Sham Neurosurgery
    Intervention: Procedure: Sham Surgery
Publications * Marks WJ Jr, Bartus RT, Siffert J, Davis CS, Lozano A, Boulis N, Vitek J, Stacy M, Turner D, Verhagen L, Bakay R, Watts R, Guthrie B, Jankovic J, Simpson R, Tagliati M, Alterman R, Stern M, Baltuch G, Starr PA, Larson PS, Ostrem JL, Nutt J, Kieburtz K, Kordower JH, Olanow CW. Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol. 2010 Dec;9(12):1164-1172. doi: 10.1016/S1474-4422(10)70254-4. Epub 2010 Oct 20.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 25, 2009)
58
Original Enrollment  ICMJE
 (submitted: November 16, 2006)
51
Actual Study Completion Date  ICMJE November 2008
Actual Primary Completion Date November 2008   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 35 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00400634
Other Study ID Numbers  ICMJE CERE-120-02
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Ceregene
Study Sponsor  ICMJE Ceregene
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Joao Siffert, M.D. Ceregene
PRS Account Ceregene
Verification Date July 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP