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Official Study Title: A Phase 1/2 Trial Assessing the Safety and Efficacy  EfficacyThe extent to which a specific intervention, procedure, or regimen produces a beneficial result under ideal conditions. of Bilateral  BilateralBoth sides of the body. Intraputaminal and Intranigral Administration of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) in Subjects With Idiopathic  IdiopathicOf, relating to, or designating a disease having no known cause. Parkinson's Disease
Sponsor: Ceregene, Inc.
Clinicaltrials.gov ID: NCT00985517
Study ID: CERE-120-09

Summary

The purpose of this study is to evaluate the safety and potential benefits of CERE-120 in the treatment of Parkinson's disease  Parkinson's DiseaseA chronic, slowly progressive disease of the nervous system characterized by the combination of tremor, rigidity, bradykinesia and stooped posture, among other symptoms. (PD). CERE-120 is an experimental drug  Experimental DrugDrug that is being tested in clinical trials prior to receiving FDA approval for use on the open market or as a treatment for a particular condition. that is designed to help damaged nerve cells in the brain function better. CERE-120 employs a virus to transfer the gene  GeneUnits of heredity that are made of DNA and contained in every cell. They produce proteins that control biological development and function. that makes neurturin, a protein that may make nerve cells in the brain healthier and protect them from dying. CERE-120 is administered during a neurosurgical procedure. The virus used in CERE-120 is not known to cause disease in people. CERE-120 has been carefully studied in laboratory animals and is in the early stages of being tested in people.

Approximately sixty people with PD will participate in this study. The first part of the study is designed to evaluate the safety of two different doses of CERE-120. Six people will participate in this part of the study, all of whom will receive CERE-120. The second part of the study will provide more information about the safety of CERE-120 and also evaluate if it is beneficial in the treatment of PD. In this portion of the study, half of the participants will receive CERE-120 and the other half will undergo a placebo  PlaceboAn inactive substance or procedure (often a pill, liquid, or powder) that has no biological effect. In clinical trials, experimental treatments are often compared with placebos to assess the treatment's effectiveness. In some studies, the participants will be assigned to a control group and will receive a placebo instead of an active drug or treatment. surgery where no medication will be injected. Participants in both phases of the study will be followed for three years after surgery.

Study Phase

Phase 1/2
What is a study phase?

Symptoms Addressed: Movement and Non-movement

General PD symptoms, Loss of Motor Skills, Postural difficulties, Rigidity,  RigidityA symptom in which muscles feel stiff and display resistance to movement even when another person tries to move the affected part of the body. Slowness of movement (bradykinesia)  BradykinesiaSlowness of movement. , Speech problems (hypophonic)  HypophonicRelating to hypophonia, or the reduction in the volume and clarity of one's voice. , Tremor,  TremorShaking or rhythmic movement, especially in the hands but in other parts of the body. Often occurs when at rest, such as in the RESTING TREMOR. Uncontrollable movements (Dyskinesia)  DyskinesiaTwisting, turning or other abnormal involuntary movement usually involving the arms, legs, trunk, and head. It is most commonly associated with long-term use of levodopa. , Walking/Gait,  GaitWay of walking, which is diminished in people with Parkinson’s who tend to shuffle their feet, take too short steps, and not swing one or both arms. Writing difficulty

Time Commitment

• More than six months
• After providing written informed consent  Informed ConsentThe process of providing information to potential study participants to help them decide whether or not to enroll in a specific clinical trial. to participate, participants in both phases of the study will enter an Eligibility Evaluation Period. Hospital stay following study surgery is usually less than 48 hours. All participants who undergo surgery will then enter a Treatment Observation Period. After study surgery, participants will return approximately every three months. In Phase 2, blinded observation will last at least 15 months. Participants will be evaluated for safety for a total of 36 months.

Eligibility

• Minimum Age: 35
• Maximum Age: 70
• Gender(s) Accepted: Either

Inclusion Criteria

• A diagnosis of idiopathic Parkinson's disease, including bradykinesia and either resting tremor  Resting TremorA tremor of a limb that increases when the limb is at rest. or rigidity
• Experiencing motor complications despite adequate antiparkinsonian therapy  TherapyAnother word for “treatment”.
• Able to walk independently
• A robust response to dopaminergic therapy as judged by the investigator
• A stable, optimized regimen of antiparkinsonian medications and stable parkinsonian features for at least 6 weeks prior to Screening  ScreeningPeriod of selection of clinical trial participants based on ELIGIBILITY CRITERIA.
• Participant is willing to NOT undergo DBS for at least 12 months after the study surgical procedure (Phase 1 participants) or while the study is blinded (Phase 2 participants) and the investigator believes that this is medically acceptable
• Participant is willing to NOT receive treatment with apomorphine IV infusions, routine administration of apomorphine injections, or Duodopa® during the course of the study - Note: Apomorphine injections may be given as rescue therapy during the study if the investigator feels this is required
• Normal coagulation (blood clotting) tests and normal platelet levels at Screening and Baseline  BaselinBeginning measurements against which a participant’s progress can be quantified at the end of a study.
• Medically fit to undergo the study surgical procedure as determined by medical history, clinical  ClinicalDealing with or based on observation and treatment of people, as opposed to basic science carried out in the laboratory or in animals. and laboratory evaluations, and any other pre-surgical evaluations that are standard at the institution where the participant will undergo surgery
• Physically and mentally capable of performing all study assessments and complying with the study visit schedule
• Participants must be able to travel to study visits alone or able to identify a partner or caregiver who agrees to accompany the participant to the study visits
• Females of childbearing potential must have a negative pregnancy test at Screening and again before surgery on Day 0
• All participants, 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

• Clinically diagnosed dementia  DementiaA decline in higher-level brain functions, such as memory, reasoning and personality. or depression
• Atypical or secondary parkinsonism, including, but not limited to, multiple system atrophy (MSA) or progressive supranuclear palsy
• Any person for whom participation in the study would pose a substantial safety risk, including, but not limited to, persons with: compromised health status that might make it difficult to achieve a quick and complete recovery from the surgical procedure or to travel to the study center, attend multiple-follow-up visits, or remain in the practically-defined "off" condition (off medication) for a sustained period of time for study assessments
• Drug-induced hallucinations or neuroleptic (anti-psychotic) treatment during the twelve months prior to Screening
• Any history of schizophrenia or other psychotic disorder
• Any major affective disorder within the 5 years prior to Screening (e.g. major depression or bipolar disorder)
• Suicidal thoughts or suicide attempt within the 5 years prior to Screening
• Drug or alcohol abuse or dependence within the 2 years prior to Screening
• Significant cardiovascular risk factors or any history of significant cardiovascular disorder
• Any disorder that would preclude surgery or alter wound healing [e.g. signs of sepsis (blood infection) or inadequately treated infection]
• Any clinically significant abnormal findings on physical examination or laboratory testing at Screening or Baseline
• Any other serious medical illness that might preclude safe participation in the study
• Any medical condition that is likely lead to disability during the course of the study and therefore confound study assessments (e.g. severe degenerative  DegenerativeGradual deterioration of organs and cells along with loss of function. joint disease, arthritis, compromised nutritional state, neuropathy, myelopathy, morbid obesity, etc.)
• Any clinical evidence of cognitive  CognitiveRelated to mental activities having to do with perception, memory, judgment, and reasoning. impairment that would confound the assessment of safety and efficacy in this trial or interfere with the ability to provide informed consent
• Any condition that would compromise the ability of the person 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 person
• 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 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 participant's well-being
• Not likely to be available for the duration of the trial, likely to be noncompliant with the protocol,  ProtocolA plan that is the basis for the conduct of a clinical trial ensuring the health of participants, and the solutions to specific research questions. A protocol describes what types of people may participate in the trial, the schedule of tests, procedures, medications and dosages, and the length of the study. or who are deemed unsuitable by the investigator for any other reason
• Participation in a previous surgical treatment study for Parkinson's disease (Applicable to Phase 2 only)
  

Enrollment

Expected Enrollment: 60 (US)
Date Enrollment Began: Sep 2009
Date Enrollment Ends: May 2011
Last Updated Date: Nov 13 2009
Trial Post Date: Nov 09 2009
Website: http://clinicaltrials.gov/ct2/show/NCT00985517

Primary Contacts and Locations

All States

• Joao Siffert, M.D. — Chief Medical Officer
  Ceregene, Inc.
  jsiffert@ceregene.com
  Phone: (858) 458-8890
  All States
  USA
  

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