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Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00985517
Recruitment Status : Completed
First Posted : September 28, 2009
Results First Posted : March 26, 2020
Last Update Posted : April 16, 2020
Sponsor:
Information provided by (Responsible Party):
Sangamo Therapeutics

Brief Summary:
The purpose of this study was to evaluate the safety and potential benefits of CERE-120 in the treatment of Parkinson's disease.

Condition or disease Intervention/treatment Phase
Idiopathic Parkinson's Disease Biological: CERE-120: Adeno-Associated Virus Delivery of Neurturin Procedure: Sham Surgery Phase 1 Phase 2

Detailed Description:

CERE-120 is an experimental drug that consists of an adeno-associated virus (AAV) that was engineered to carry the human gene for neurturin, a neurotrophic (growth) factor. Similar to other growth factors (such as GDNF), neurturin is capable of restoring function and protecting brain cells from further damage. The virus used in CERE-120 is not known to cause disease in people.

CERE-120 is delivered directly to the brain cells most affected in Parkinson's disease - the dopamine producing neurons. CERE-120 is injected during brain surgery. Once in place, CERE-120 continuously produces neurturin.

During the first, open-label, part of the study (Phase 1), subjects with Parkinson's disease received CERE-120 at one of two dose levels. In the second part of the study (Phase 2), subjects were randomized 1:1 to receive CERE-120 or a "sham" surgery where no medication was injected.

Participants in both phases of the study were followed for up to five years after surgery.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 57 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

In the first part of the study (Phase 1), 2 escalating dose cohorts received CERE-120.

In the second part of the study (Phase 2), subjects were randomized 1:1 to receive CERE-120 or Sham Surgery.

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Trial Assessing the Safety and Efficacy of Bilateral Intraputaminal and Intranigral Administration of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) in Subjects With Idiopathic Parkinson's Disease
Actual Study Start Date : October 29, 2009
Actual Primary Completion Date : November 9, 2014
Actual Study Completion Date : November 16, 2017

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Phase 1: Cohort 1
CERE-120 9.4 x 10^11 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen
Biological: CERE-120: Adeno-Associated Virus Delivery of Neurturin
Other Name: AAV2-Neurturin

Experimental: Phase 1: Cohort 2
CERE-120 2.4 x 10^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen
Biological: CERE-120: Adeno-Associated Virus Delivery of Neurturin
Other Name: AAV2-Neurturin

Experimental: Phase 2: CERE-120
CERE-120 2.4 x 10^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen
Biological: CERE-120: Adeno-Associated Virus Delivery of Neurturin
Other Name: AAV2-Neurturin

Sham Comparator: Phase 2: Sham Surgery
Neurosurgical procedure that mimics the procedure for CERE-120 delivery. No injections were administered during sham surgery.
Procedure: Sham Surgery



Primary Outcome Measures :
  1. Number of Participants Who Received CERE-120 Treatment [ Time Frame: 5 years ]
    Number of Participants who received CERE-120 Treatment

  2. Phase 1 and Phase 2: Change From Baseline in Motor Examination Part III Total Score in the "Off" Condition for the CERE-120 Group as Compared to the Sham Surgery Control Group at the Last Double-blind Assessment. [ Time Frame: Baseline, Months 15, 18, 21 and 24 ]

    UPDRS is a tool to evaluate the impact of symptomatic and potential disease modifying treatments. Part III focuses on 14 motor functions, assessing each on a scale from 0 (normal) to 4 (severe) with total score 0 - 56. The 5 Feb 2003 version of the UPDRS Rating was used in this trial. The total score is the sum of 14 motor functions. Change from baseline in total score is reported.

    The last double blind assessment for each subject is defined as the most recent assessment at the time when the final randomized subject completes the Month 15 Visit. The length of the double-blind follow-up period for each subject ranged from 15 to 24 months, depending on the enrollment rate.




Information from the National Library of Medicine

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Ages Eligible for Study:   35 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males and females, ages 35 to 70 years old (inclusive)
  • A diagnosis of idiopathic Parkinson's disease based on UK Brain Bank criteria, including bradykinesia and at least 1 of the following PD features: resting tremor or rigidity
  • A Hoehn and Yahr score of no greater than 3 in the "off" condition at Screening
  • A robust response to dopaminergic therapy as judged by the investigator based on the UPDRS Part III: Motor Examination
  • Experiencing motor complications despite adequate antiparkinsonian therapy
  • A stable, optimized regimen of antiparkinsonian medications and stable parkinsonian features for at least 6 weeks prior to Screening
  • Subject is willing not to undergo DBS for at least 12 months after the study surgical procedure (Phase 1 subjects) or while the study is blinded (Phase 2 subjects) and the investigator believes that this is medically acceptable
  • Medically fit to undergo the study surgical procedure as determined by medical history, clinical and laboratory evaluations, and any other pre-surgical evaluations that are standard at the institution where the subject will undergo surgery
  • Physically and mentally capable of performing all protocol-specified assessments and complying with the study visit schedule
  • Subjects must be able to travel to study visits alone or able to identify a partner or caregiver who agrees to accompany the subject to the study visits
  • Females of childbearing potential must have a negative β-HCG pregnancy test at Screening and again before surgery on Day 0
  • All subjects, both male and female, must agree to practice adequate barrier method contraception for at least 6 months after the surgical procedure
  • Provides written informed consent to participate before any study-specific procedures are conducted

Exclusion Criteria:

  • Atypical or secondary parkinsonism, including, but not limited to, multiple system atrophy (MSA) or progressive supranuclear palsy
  • Any subject for whom participation in the study would pose a substantial safety risk
  • Any condition that would compromise the ability of the subject to undergo study procedures, including allergy to gadolinium
  • Presence of any known brain abnormality that could interfere with the assessment of safety or efficacy or represents a surgical risk to the subject
  • Evidence of significant brain atrophy on the Baseline MRI
  • History of any cancer other than basal or squamous cell skin cancer within the 3 years prior to Screening
  • Any chemotherapy, cytotoxic therapy, or immunotherapy (e.g., IL-2, IL-12, interferon) within the 3 months prior to Screening
  • Any prior treatment for PD with a procedure involving intracranial surgery or implantation of a device (e.g. DBS, pallidotomy)
  • Any prior treatment for a neurological or psychiatric disorders with a procedure involving the implantation of a device (e.g. spinal cord stimulator, vagus nerve stimulator)
  • History of any prior gene transfer therapy
  • Treatment with any investigational agent within the 3 months prior to Screening
  • Anticipated need for antiplatelet agents or anticoagulation therapy, including gingko biloba, during the 10 days prior to the projected surgery date
  • Any vaccinations within the 30 days prior to the projected surgery date Note: Vaccinations are not allowed for 30 days after the surgical procedure, unless deemed necessary by the investigator for the subject's well-being
  • Not likely to be available for the duration of the trial, likely to be noncompliant with the protocol, or who are deemed unsuitable by the investigator for any other reason
  • Participation in a previous surgical treatment study for Parkinson's disease

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00985517


Locations
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United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States
United States, California
Stanford School of Medicine
Palo Alto, California, United States
University of California, San Francisco
San Francisco, California, United States
United States, Georgia
Emory University
Atlanta, Georgia, United States
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States
United States, New York
Beth Israel Medical Center
New York, New York, United States
Columbia University Medical Center
New York, New York, United States
Mount Sinai Medical Center
New York, New York, United States
United States, North Carolina
Duke University
Durham, North Carolina, United States
United States, Pennsylvania
University of Pennsylvania
Philadelphia, Pennsylvania, United States
United States, Texas
Baylor College of Medicine
Houston, Texas, United States
Sponsors and Collaborators
Sangamo Therapeutics
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Sangamo Therapeutics
ClinicalTrials.gov Identifier: NCT00985517    
Other Study ID Numbers: CERE-120-09
First Posted: September 28, 2009    Key Record Dates
Results First Posted: March 26, 2020
Last Update Posted: April 16, 2020
Last Verified: April 2020
Keywords provided by Sangamo Therapeutics:
Parkinson's disease
Movement Disorders
Gene Therapy
Neurotrophic Factors
GDNF
Neurturin
DBS
Dopamine
Additional relevant MeSH terms:
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Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases