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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00985517
• Date of registration: 25/09/2009
• Prospective Registration: Yes
• Primary sponsor: Sangamo Therapeutics
• Public title: Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease
• Scientific 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
• Date of first enrolment: October 29, 2009
• Target sample size: 57
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00985517
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Key inclusion & exclusion 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 ß-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



Age minimum: 35 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Idiopathic Parkinson's Disease

Intervention(s)

Biological: CERE-120: Adeno-Associated Virus Delivery of Neurturin

Procedure: Sham Surgery

Primary Outcome(s)

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]

Number of Participants Who Received CERE-120 Treatment [Time Frame: 5 years]

Secondary ID(s)

CERE-120-09

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 26/03/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00985517