for people ages 35-70 (full criteria)
at San Francisco, California and other locations
study started
estimated completion:
Jill Ostrem



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

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


Approximately sixty patients with Parkinson's disease will participate in this study. The first part of the study (Phase 1) is now complete. Six people with Parkinson's disease received CERE-120 in Phase 1 without complications.

The second part of the study (Phase 2) will provide more information about the safety of CERE-120 and also evaluate if it is beneficial in the treatment of Parkinson's disease. In Phase 2, approximately 52 people with Parkinson's disease will be enrolled. Half of the subjects will receive CERE-120 and the other half will undergo a "placebo" surgery where no medication will be injected. Treatment assignment will be random (like the flip of a coin). Participants in both phases of the study will be followed for three years after surgery.

In this study, CERE-120 will be injected by a neurosurgeon directly in the substantia nigra (where dopamine producing cells are located) and in the putamen (where the dopamine cells project).

CERE-120 has been carefully studied in laboratory animals without any toxicity despite very high doses. In addition, in animals with symptoms similar to Parkinson's disease, CERE-120 was shown to protect brain cells and restore their function.

CERE-120 has been previously studied in 50 people with Parkinson's enrolled in two clinical studies. These patients have been followed for over 2 years (some for almost 5 years) and so far CERE-120 has been well tolerated. Whereas CERE-120 was not better than placebo on the primary efficacy measure in a completed study, several measures of improvement suggested a modest benefit. In order to build upon these findings and increase the chances for a stronger clinical benefit, a dose increase and modifications in the delivery targets have been implemented in the current study.


Idiopathic Parkinson's Disease Parkinson's disease Movement Disorders Gene Therapy Neurotrophic Factors GDNF Neurturin DBS Dopamine Parkinson Disease CERE-120: Adeno-Associated Virus Delivery of Neurturin Sham Surgery CERE-120


You can join if…

Open to people ages 35-70

  • 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 CAN'T join 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


  • University of California, San Francisco
    San Francisco California United States
  • Stanford School of Medicine
    Palo Alto California United States

Lead Scientist

  • Jill Ostrem
    Professor, Neurology. Authored (or co-authored) 91 research publications


in progress, not accepting new patients
Start Date
Completion Date
Sangamo Therapeutics
Phase 1/2
Study Type
Last Updated