GDNF in ideopathicParkinsons Disease
Condition: Idiopathic Parkinson Disease
MeSH terms: Parkinson Disease
Study type: Interventional
Phase: Phase 2

Summary: 
      A Placebo Controlled Randomised Trial of GDNF vs placebo

      The study will require patients to undergo surgery to implant microcatheters precisely into
      the brain. Patients will then attend clinic on a 2 weekly basis for infusions of a nerve
      growth factor called GDNF or placebo. Specific tests will also be carried out at regular
      intervals to assess your symptoms. All participants will undergo radio-isotope brain imaging
      at the beginning and end of the study. Periodically patients will also be required to undergo
      an MRI scan to assess the delivery of the study drug or placebo.
    

Detailed description: 
      Patients who are considered likely to meet certain suitability criteria for this trial and
      who provide fully informed consent to participate will have screening blood tests and
      clinical assessments. As part of these assessments patients will be asked to stop their PD
      medication overnight to allow us to assess the severity of their PD symptoms without the
      benefit of medication on board. These assessments will answer if patients are eligible to
      enter the trial. Some patients will not continue on with the trial following these
      assessments. However, we will discuss with those patients alternative clinical care /
      research options.

      Patients that are felt to be eligible for the trial following these assessments will undergo
      brain surgery performed under general anaesthetic. During the surgery 4 tiny plastic infusion
      tubes will be inserted into the putamen area of the brain, 2 on either side of the brain (two
      in each putamen). These tubes connect to a single metal port that will come out (exit) from
      under the skin behind the ear. This port can be accessed as needed from the outside and will
      allow GDNF or placebo infusions to be given every 4 weeks directly into the putamen part of
      the brain without requiring additional surgery or piercing of the skin (see Figure 1).

      Approximately 4 weeks after surgery patients will have an infusion of placebo / dummy drug
      and an MRI to check the tubes and port system are working well enough to allow infusions of
      either GDNF or placebo (further details below).

      Patients will be required to attend NBT in order to have either GDNF or placebo infusions
      given through the port and down the tiny plastic tubes into the putamen every 4 weeks for a
      total of 9 months (10

      infusions in total). Neither the patient nor the research team will know whether patients are
      receiving GDNF or placebo / dummy infusions.

      For some of these visits, patients will again be asked to stop their PD medication the night
      before. By doing this, it will be possible to assess potential reversal of symptoms compared
      with before the start of brain infusion treatments.During the course of this trial patients
      will undergo positron emission tomography (PET) brain scans at another hospital on two or
      three occasions to look for evidence of damaged brain cell recovery. This trial is therefore
      time-consuming and this will be made clear to you before seeking your consent to participate.

      We hope that this trial will help clarify whether GDNF does indeed have the potential to
      reverse Parkinson's disease and restore parts of the brain. It must be understood, however,
      that even if the trial yields positive results, further testing will be required in a larger
      number of patients to meet the requirements of the health authorities prior to GDNF being
      available over the long term or routinely.

      Participation in this trial will involve some risks to patients. These risks are described in
      detail later in this document and will be discussed with you before you give informed
      consent. When the trial ends the information collected from participants will be used to
      produce reports for scientific journals, and will be presented at scientific meetings so that
      doctors and scientists around the world will better understand the potential brain restoring
      and disease reversing effects of GDNF in PD.
    

Intervention name(s): glial cell line-derived neurotrophic factor
Intervention type(s): Drug
Intervention description(s): Intermittent Bilateral Intraputamenal Glial Cell Line-Derived Neurotrophic Factor (GDNF) Infusions Administered via Convection Enhanced Delivery
Other intervention name(s): GDNF
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment