The trial (NCT03295786) is testing the safety and tolerability of CDNF in 17 patients with advanced Parkinson’s disease. Of these patients, 15 completed the main study and will advance into a six-month extension study where they will continue to receive a monthly dose of CDNF. Two patients left the study for reasons not related to the treatment.
“We are very grateful to the patients, the investigators, and the hospital staff of this clinical study,” Pekka Simula, CEO of Herantis, said in a press release. “We look forward to the excellently continuing collaboration in the ongoing extension study, as well as to the near-future read-outs from the main study.”
The trial began at the Karolinska University Hospital, Sweden, and then was extended to two other clinical sites, Lund University Hospital, also in Sweden, and the Helsinki University Hospital in Finland.
CDNF is based on a protein naturally present in the blood and cerebrospinal fluid (CSF) — the liquid surrounding the brain and spinal cord. Preclinical studies have shown it has neuroprotective and neurorestorative properties in brain cells that generate the neurotransmitter dopamine, which is the chemical messenger missing in people with Parkinson’s disease.
Because CDNF cannot be delivered as a pill or injection — since the body will not transport it to the brain — a neurosurgeon needs to implant a drug delivery system in patients’ brains.
During the trial, participants were randomly assigned to receive monthly infusions of either CDNF (mid- or high-dose) or a placebo for six months.
The study’s primary objectives included the tolerability and safety of CDNF and its delivery device as well as the accuracy of surgical placement of the device.
Additional goals include CDNF’s early signs of efficacy, including its effect on the Unified Parkinson’s Disease Rating Scale motor score, patient-reported outcomes, and the levels of different forms of alpha-synuclein in the blood and CSF.
All 15 patients who completed the first part of the study opted to join the extension phase in which all of them (including those previously randomized to the placebo) will receive six doses of CDNF over a period of six months. All patients will start on a low dose, which can then be increased after an independent group of experts confirms there are no safety concerns associated with the therapy.
The extension study is expected to be completed by the end of 2020, and will be followed by a long-term follow-up. Herantis expects to share the results from this first group of patients by the first quarter of this year.
In addition, Herantis launched a program in 2018 to develop a noninvasive administration of CDNF that will retain the therapy’s potential but is easier to administer.