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