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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03295786
• Date of registration: 14/09/2017
• Prospective Registration: Yes
• Primary sponsor: Herantis Pharma Plc.
• Public title: Clinical Study to Test the Safety of CDNF by Brain Infusion in Patients With Parkinson's Disease
• Scientific title: Phase 1-2, Randomised, Double-Blind, Placebo Controlled, Safety and Tolerability Study of Intraputamenal Cerebral Dopamine Neurotrophic Factor (CDNF) Infusions Via an Investigational Drug Delivery System to Patients With Parkinson's Disease
• Date of first enrolment: September 26, 2017
• Target sample size: 17
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03295786
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

Finland, Sweden

Contacts

• Name: Per Svenningsson, MD
• Affiliation: Karolinska University Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Idiopathic Parkinson's disease based on UK brain bank criteria

2. Duration of PD motor symptoms 5-15 years (inclusive)

3. Age 35-75 years (inclusive)

4. Presence of motor fluctuations.

5. At least 5 daily doses of levodopa

6. Ability to reliably distinguish motor states and accurately complete fluctuation
diaries

7. UPDRS motor score (part III) in a practically defined OFF-state between 25-50
(inclusive)

8. Hoehn and Yahr = stage III in the OFF-state

9. Responsiveness to levodopa

10. No change in anti-parkinsonian medication for 6 weeks before screening

11. Provision of Informed Consent

Exclusion Criteria:

1. Diagnosed with atypical parkinsonism or any known secondary parkinsonian syndrome.

2. Signs or symptoms suggestive of atypical parkinsonian syndrome.

3. Drug-resistant rest tremor.

4. Prior neurosurgical treatment for PD, including lesioning or deep brain stimulation

5. Significant neurological disorder other than PD including clinically significant head
trauma, cerebrovascular disease, epilepsia, CSF shunt or other implanted CNS device

6. Presence of significant depression as defined as a BDI score = 20

7. Current psychosis requiring therapy.

8. Presence of clinically significant impulse control disorder ((QUIP-RS) score > 20),
or, presence of dopamine dysregulation syndrome.

9. MoCA score < 24.

10. Use within 3 months of planned catheter insertion of concomitant medications known to
affect PD symptoms other than prescribed PD therapy.

11. Any medical condition, which might impair outcome measure assessments or safety
measures including ability to undergo MRI or DAT-PET.

12. Hypersensitivity or allergy to gadolinium or to any of excipients of macrocyclic GBCA
used for the surgical planning MRI.

13. Screening and/or planning MRI demonstrating any abnormality, which would suggest an
alternative cause for patient's parkinsonism or preclude neurosurgery.

14. 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

15. History within the last 5 years of cancer with the exception of basal cell carcinoma
of the skin

16. History of drug or alcohol abuse within 2 years of screening

17. Use of any investigational drug or device within 90 days of screening

18. Active breastfeeding



Age minimum: 35 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Nervous System Diseases

Brain Diseases

Movement Disorders

Parkinson Disease

Neurodegenerative Diseases

Intervention(s)

Drug: Cerebral Dopamine Neurotrophic Factor

Device: Renishaw Drug Delivery System

Primary Outcome(s)

Beck Depression Inventory (BDI) score [Time Frame: Week 15 to Week 40]

Device related accuracy of implantation [Time Frame: Week 8]

Device related changes in safety measures [Time Frame: Week 8 to Week 40]

Electrocardiogram (ECG) [Time Frame: Week 15 to Week 40]

Adverse events (AEs) [Time Frame: Week 15 to Week 40]

Montreal cognitive assessment (MoCA) [Time Frame: Week 15 to Week 40]

Questionnaire for impulsive-compulsive disorder in Parkinson's disease rating scale (QUIP_RS) [Time Frame: Week 15 to Week 40]

Formation of anti-CDNF antibodies [Time Frame: Week 15 to Week 40]

Clinical laboratory safety screen [Time Frame: Week 15 to Week 40]

Physical examination [Time Frame: Week 15 to Week 40]

Vital signs [Time Frame: Week 15 to Week 40]

Secondary Outcome(s)

PDQ-39 (Parkinson's Disease Questionnaire) score [Time Frame: Week 15 to Week 40]

change in CGI (Clinical Global Impressions) scale [Time Frame: Week 16 to Week 40]

TUG (Timed Up and Go) test [Time Frame: Week 15 to Week 40]

Cessation of infusions [Time Frame: Week 11 to Week 36]

Home diary score [Time Frame: Week 16 to Week 24]

Occurrence of blockage [Time Frame: Week 11 to Week 36]

UPDRS (Unified Parkinson's Disease Rating Scale) Part III motor score [Time Frame: Week 15 to Week 40]

UPDRS Total score (Part I-IV) [Time Frame: Week 15 to Week 40]

Secondary ID(s)

HP-CD-CL-2002

2015-004175-73

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Renishaw plc.