Safety of CDNF by Brain Infusion in Patients With Parkinson's Disease. Extension to HP-CD-CL-2002 Clinical Study

A Randomised, Double-Blind, Multi-centre, Active Treatment, Extension and Safety Study for Patients With Idiopathic Parkinson's Disease (PD) Who Previously Completed the CDNF/DDS Main Study HP-CD-CL-2002

This study is an extension to the HP-CD-CL-2002 clinical study. It evaluates the long-term safety and tolerability of CDNF in patients with Parkinson's disease when dosed directly into the brain using an implanted investigational drug delivery system (DDS). Long-term safety of the DDS is also being evaluated. All patients will receive monthly infusions of either mid- or high-dose of CDNF for a period of 6 months.

A patient's participation in the study will last for six months and will include nine visits:

Screening (1 visit, same as HP-CD-CL-2002 End-of-Study visit) Dosing visits: CDNF (6 visits) DAT-PET (1 visit) End-of-study visit (1 visit)

Study examinations and assessments

• Physical examination: pulse rate, blood pressure, temperature, body weight and height, body mass index (BMI), neurological exam
• ECG (electrocardiography) and blood and urine tests
• 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 movement recording device
• 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)

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

Inclusion Criteria:

1. Completion of 6 months treatment period in the Main study (HP-CD-CL-2002) including End-of-Study assessment
2. Negative pregnancy test at study entry for females of childbearing potential. Willingness of using a highly effective form of contraception until 30 days after end of study. Males: willingness to use condom and not to donate sperm for 3 months following DAT-PET. Willingness of female partners of male study participants to use highly effective form of contraception until 30 days after their male partner's end of the study.
3. At least one functioning catheter in each putamen
4. Provision of informed consent

Exclusion Criteria:

1. Drug-resistant rest tremor, severe dyskinesia or severe head tremor, which could interfere with treatment infusions
2. Significant neurological disorder other than PD including clinically significant head trauma, cerebrovascular disease, epilepsy, CSF shunt or other implanted central nervous system device
3. Changes in pathology which give rise to safety concern such as sequelae from catheter implantation, clinically significant intracerebral trauma, oedema, haemorrhage, or infection
4. Current psychosis requiring therapy
5. Presence of clinically significant impulse control disorder by a positive screen on the QUIP-RS questionnaire score >20, or, presence of dopamine dysregulation syndrome
6. An unresolved intolerable adverse event or adverse device event in study HP-CD-CL-2002, which is not expected to resolve or cease to an acceptable level of intensity within reasonable time
7. Medical conditions, which might impair outcome measure assessments or safety measures
8. Impaired renal function
9. Concomitant treatment with neuroleptics or antipsychotic medication prescribed for treatment of current psychosis, central dopamine blockers or tricyclic antidepressants