An Open-Label Pilot Study for Assessing the Safety and Efficacy of High-Dose Ambroxol (HDA) in Newly Diagnosed GBA1 Parkinson Disease (PD)
Parkinson's disease (PD), affecting 10 million people globally, lacks a cure, and current therapies only manage symptoms. A link between Gaucher disease (GD) and PD, particularly in carriers of glucocerebrosidase (GBA1) mutations, has sparked interest in developing new drugs. Despite pharmaceutical companies focusing on formulations, progress is slow. Agyany, with decades of experience in GD research, plans clinical trials using existing generic drugs for GBA-related PD and idiopathic PD. Their approach targets the misfolded enzyme glucocerebrosidase with pharmacological chaperons, inspired by success in GD using ambroxol. The strategy aims to provide a quicker path to novel therapeutic options for PD.
∙ For inclusion into the trial, subjects are required to fulfill all of the following criteria:
∙ Newly diagnosed PD patients:
• Individuals who exclusively carry at least one single GBA1 variant without any additional genetic variants.
• Confirmed diagnosis of PD, by a movement disorder specialist, according to MDS PD criteria, within a maximum of three years from the date of diagnosis, coupled with the following conditions:
• iii. Hoehn and Yahr staged between I-II, inclusive.
• iv. No motor fluctuations or L-dopa induced dyskinesia.
• Stable anti-PD medications for ≥ 4 weeks:
• Subjects can take PD medications including NMDA glutamate antagonists, monoamine oxidase B (MAO-B) inhibitors, dopamine agonists, and L-Dopa.
• Male or female, age 30-70 years; however, if female:
‣ must be using contraception measures if of childbearing potential.
⁃ must not be lactating.
• Complying with study protocol.