Clinical Study to Test the Safety of CDNF by Brain Infusion in Patients With Parkinson's Disease

A patient's participation in the study will last for ten months and will include sixteen to seventeen visits:

• Screening (2 visits)
• Planning of surgery - Surgery: implantation of drug delivery system - Post-surgery follow-up (3 visits)
• Test infusions with vehicle (1-2 visits)
• Positron emission tomography (PET) examinations before the first and after the last dose (2 visits)
• Baseline and randomisation to CDNF or placebo group (1 visit)
• Dosing visits: CDNF or placebo (6 visits)
• End-of-study visit (1 visit)

Study examinations and assessments

• Physical examination: pulse rate, blood pressure, temperature, body weight and height

• ECG (electrocardiography) and blood and urine tests
• HIV, hepatitis B and C blood tests (on first visit)
• Pregnancy tests for women of childbearing age
• Completion of a patient diary to record mobility and time asleep
• Parkinson's Kinetigraph (PKGTM) Data Logger: a watch-type device worn on the wrist for certain periods during the study to record movements
• Questionnaires, rating scales and forms: quality of life, mood, memory, impulse control, mental health
• Assessment of the port and the skin around the port
• Cerebrospinal fluid sampling by lumbar puncture
• Magnetic resonance imaging (MRI)
• Positron emission tomography scans (PET)
• Computed tomography (CT)

For more information: https://treater.eu/clinical-study/

Inclusion criteria

• Idiopathic Parkinson's disease based on UK brain bank criteria
• Duration of PD motor symptoms 5-15 years (inclusive)
• Age 35-75 years (inclusive)
• Presence of motor fluctuations.
• At least 5 daily doses of levodopa
• Ability to reliably distinguish motor states and accurately complete fluctuation diaries
• UPDRS motor score (part III) in a practically defined OFF-state between 25-50 (inclusive)
• Hoehn and Yahr ≤ stage III in the OFF-state
• Responsiveness to levodopa
• No change in anti-parkinsonian medication for 6 weeks before screening
• Provision of Informed Consent

Exclusion criteria

• Diagnosed with atypical parkinsonism or any known secondary parkinsonian syndrome.
• Signs or symptoms suggestive of atypical parkinsonian syndrome.
• Drug-resistant rest tremor.
• Prior neurosurgical treatment for PD, including lesioning or deep brain stimulation
• Significant neurological disorder other than PD including clinically significant head trauma, cerebrovascular disease, epilepsia, CSF shunt or other implanted CNS device
• Presence of significant depression as defined as a BDI score ≥ 20
• Current psychosis requiring therapy.
• Presence of clinically significant impulse control disorder ((QUIP-RS) score > 20), or, presence of dopamine dysregulation syndrome.
• MoCA score < 24.
• Use within 3 months of planned catheter insertion of concomitant medications known to affect PD symptoms other than prescribed PD therapy.
• Any medical condition, which might impair outcome measure assessments or safety measures including ability to undergo MRI or DAT-PET.
• Hypersensitivity or allergy to gadolinium or to any of excipients of macrocyclic GBCA used for the surgical planning MRI.
• Screening and/or planning MRI demonstrating any abnormality, which would suggest an alternative cause for patient's parkinsonism or preclude neurosurgery.
• Any medical condition that would put the patient at undue risk from surgical treatment or chronic implants including but not limited to bleeding disorders, chronic infections, or immunosuppressive illness
• History within the last 5 years of cancer with the exception of basal cell carcinoma of the skin
• History of drug or alcohol abuse within 2 years of screening
• Use of any investigational drug or device within 90 days of screening
• Active breastfeeding