NCT04691661

Safety, Tolerability, Pharmacokinetics and Efficacy Study of Radotinib in Parkinson's Disease

A Randomized Double-blind Placebo-controlled Multicentre Study to Assess Safety, Tolerability, Pharmacokinetics and Efficacy of Radotinib in Parkinson's Disease

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Sponsor: Il-Yang Pharm. Co., Ltd. · Industry
• Sex: All
• Age: 40 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This is a safety, tolerability, pharmacokinetic and efficacy study in subjects with Parkinson's disease

Detailed description

This study is will be conducting to determine if Radotinib is safe and can be tolerated by patients with Parkinson's disease (PD) and to learn if Radotinib can be potential therapeutic agents for the treatment of PD. Radotinib has been approved by Ministry of Food \& Drug Safety of Korea to treat Chronic Myeloid Leukemia (CML) but it has not been approved for PD. In nonclinical efficacy study, therapeutic effect of Radotinib HCl, c-Abl inhibitor, which exhibits improved pharmacokinetic properties and BBB penetration compared to nilotinib and other c-Abl inhibitors, was tested in a preclinical α-synuclein preformed fibrils (PFF) model of sporadic PD. As a result, the treatment of Radotinib HCl protects the α-synuclein PFFs-induced neuronal toxicity, reduces the PFFs-induced LB/LN-like pathology, and inhibits the PFFs-induced c-Abl activation in neurons. In vivo studies demonstrate that administration of Radotinib HCl prevents dopamine neuron loss and behavioral deficits following α-synuclein PFFs-induced toxicity. Taken together, these findings indicate that Radotinib HCl has beneficial neuroprotective effects in PD and provides strong evidence that selective and brain permeable c-Abl inhibitors can be potential therapeutic agents for the treatment of PD. These data are very compelling to evaluate the effects of Radotinib in a phase II, randomized, double-blind, placebo-controlled trial in patients with PD.

Conditions

• Parkinson Disease

Interventions

Type	Name	Description
DRUG	Radotinib HCl 50 mg	Enrolled subject will continue to administer Radotinib 50mg/day, 100mg/day, 150mg/day, 200mg/day, depending on the dose level once daily for 6 months.
DRUG	Placebo	Placebo

Timeline

• Start date: 2021-09-09
• Primary completion: 2025-12-31
• Completion: 2026-12-31
• First posted: 2020-12-31
• Last updated: 2024-07-16

Locations

7 sites across 1 country: France