Clinical study

Clinical study of CDNF treatment for Parkinson’s disease

Study title: A Phase I-II, Randomised, Double-Blind, Placebo Controlled, Safety and Tolerability Study of Intermittent Bilateral Intraputamenal Cerebral Dopamine Neurotrophic Factor (CDNF) Infusions Administered via an Investigational Drug Delivery System to Patients with Idiopathic Parkinson’s Disease (PD) of Moderate Severity.

Treatment with CDNF (Cerebral Dopamine Neurotrophic Factor) aims to modify the progression of Parkinson’s disease and to provide symptomatic relief. However, as this is a first-in-human study, the primary objectives are safety and tolerability.

Study sponsor

Herantis Pharma Plc www.herantis.com in collaboration with Renishaw Plc www.renishaw.com.

Study sites

Karolinska University Hospital, Department of Clinical Neuroscience, Stockholm, Sweden

Coordinating Investigator: Professor Per Svenningsson, specialist physician in neurology

Two other sites in Sweden and Finland plan to join the study at a later stage.

Regulatory and ethics approvals

The Swedish regulatory authority, MPA (Medical Products Agency), and the regional ethics committee in Stockholm have approved this study.

The study drug and the drug delivery system

The study drug is called CDNF (Cerebral Dopamine Neurotrophic Factor). It is a protein, which is found naturally in the body. In this study, its potential to prevent dopamine neuron degeneration and to stimulate neuronal regeneration are being studied. As CDNF is a protein, it cannot be given as a pill or an injection as the body will not transport it to the brain. Therefore, in this study, CDNF will be given using a drug delivery system, which will be implanted into the brain by a neurosurgeon.

The CDNF drug is delivered to the brain via catheters, which will be implanted by neurosurgeons in the putamen area of the brain at the beginning of the study. The catheters are connected to a port, which is fixed to the head, just behind the ear. The system also includes filters, which prevent bacteria and air from entering the brain.The investigational drug delivery system has been previously implanted in more than forty patients in another clinical study.

Figure 1. Drug delivery device used in the study. (A) Location of different components after implantation surgery are shown. (B) Close-up view of the port structure. (C) Device in dosing mode, connected to the application set for drug dosing. List of components: 1 = transcutaneous skull-anchored port, 2 = subcutaneous lines connecting the port to the catheters, 3 = catheter entry sites (x4), the brain-dwelling parts of catheters are not shown, 4 = application set attached to the port [external infusion lines (not shown) will be connected to the application set for dosing].

How will the study be conducted?

  • CDNF will be compared to placebo. Two out of three patients will receive CDNF and one out of three will receive placebo.

  • All patients who participate in the main study will be offered an opportunity to participate in an extension study, in which all patients will receive additional six doses of CDNF over a further period of six months (regulatory approval for the extension study is pending).

  • Neurological, laboratory and brain imaging assessments are included in the study for the evaluation of safety, tolerability and efficacy of the study drug and the investigational drug delivery system.