Ignite Creation Date:
2025-12-24 @ 12:13 PM
Ignite Modification Date:
2026-01-01 @ 10:44 PM
Study NCT ID:
NCT04691661
Status:
RECRUITING
Last Update Posted:
2024-07-16
First Post:
2020-12-16
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety, Tolerability, Pharmacokinetics and Efficacy Study of Radotinib in Parkinson's Disease
Sponsor:
Il-Yang Pharm. Co., Ltd.
Organization:
Study Overview
Official Title:
A Randomized Double-blind Placebo-controlled Multicentre Study to Assess Safety, Tolerability, Pharmacokinetics and Efficacy of Radotinib in Parkinson's Disease
Status:
RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: