Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease
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
1 other identifier
interventional
57
1 country
11
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Oct 2009
Longer than P75 for phase_1
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 25, 2009
CompletedFirst Posted
Study publicly available on registry
September 28, 2009
CompletedStudy Start
First participant enrolled
October 29, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 9, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
November 16, 2017
CompletedResults Posted
Study results publicly available
March 26, 2020
CompletedApril 16, 2020
April 1, 2020
5 years
September 25, 2009
September 17, 2019
April 7, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Number of Participants Who Received CERE-120 Treatment
Number of Participants who received CERE-120 Treatment
5 years
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.
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.
Baseline, Months 15, 18, 21 and 24
Study Arms (4)
Phase 1: Cohort 1
EXPERIMENTALCERE-120 9.4 x 10\^11 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen
Phase 1: Cohort 2
EXPERIMENTALCERE-120 2.4 x 10\^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen
Phase 2: CERE-120
EXPERIMENTALCERE-120 2.4 x 10\^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen
Phase 2: Sham Surgery
SHAM COMPARATORNeurosurgical procedure that mimics the procedure for CERE-120 delivery. No injections were administered during sham surgery.
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
University of Alabama at Birmingham
Birmingham, Alabama, United States
Stanford School of Medicine
Palo Alto, California, United States
University of California, San Francisco
San Francisco, California, United States
Emory University
Atlanta, Georgia, United States
Rush University Medical Center
Chicago, Illinois, United States
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
Duke University
Durham, North Carolina, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Baylor College of Medicine
Houston, Texas, United States
Related Publications (1)
Bartus RT, Baumann TL, Siffert J, Herzog CD, Alterman R, Boulis N, Turner DA, Stacy M, Lang AE, Lozano AM, Olanow CW. Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology. 2013 Apr 30;80(18):1698-701. doi: 10.1212/WNL.0b013e3182904faa. Epub 2013 Apr 10.
PMID: 23576625DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Sangamo Therapeutics
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 25, 2009
First Posted
September 28, 2009
Study Start
October 29, 2009
Primary Completion
November 9, 2014
Study Completion
November 16, 2017
Last Updated
April 16, 2020
Results First Posted
March 26, 2020
Record last verified: 2020-04