Study Rationale: sHER-096 is a novel compound that mimics CDNF, a human neurotrophic factor that has shown neuroprotective and neurorestorative properties in an animal model of Parkinson’s disease (PD). HER-096 can penetrate the blood-brain barrier, and single doses were found to be safe and well-tolerated in healthy human subjects. This project will test the safety and tolerability of repeated subcutaneous injections of HER-096 in people with PD. The project will also generate new data on biomarkers to follow how the body responds to HER-096 injections.
Hypothesis: We hypothesize that repeated subcutaneous injections of HER-096 will be safe and well-tolerated in people with PD.
Study Design: For this Phase 1b clinical study, we will recruit 24 people with PD who will be randomized to receive either placebo injections or injections with 200 or 300 mg of HER-096 two times a week for four weeks. We will closely monitor the participants for adverse events based on clinical, blood chemistry and local injection site observations. We will collect samples of blood and cerebrospinal fluid for exploratory biomarker work, with an aim to identify fluid-based biomarkers indicative of how the human body responds to HER-096 injections.
Impact on Diagnosis/Treatment of Parkinson’s Disease: The therapeutics currently available for people with PD only treat disease symptoms. HER-096 has the potential to become a disease-modifying treatment that will slow PD progression and support functional recovery of stressed and degenerating dopamine neurons.
Next Steps for Development: The next step in developing HER-096 will be a Phase 2 clinical study with longer dosing period and larger group of subjects. The Phase 2 study will generate first data on efficacy of HER-096 in people with PD.
Trial Phase: Phase 1b