Ignite Creation Date:
2024-05-06 @ 7:53 PM
Last Modification Date:
2024-10-26 @ 3:16 PM
Study NCT ID:
NCT06177431
Status:
RECRUITING
Last Update Posted:
2024-04-15
First Post:
2023-12-11
Brief Title:
An Open Label Extension Study of Monepantel in Individuals With Motor Neurone Disease
Sponsor:
PharmAust Ltd
Organization:
PharmAust Ltd
Study Overview
Official Title:
An Open Label Extension Study to Investigate the Long Term Safety Tolerability And Efficacy of Oral Monepantel in Individuals With Motor Neurone Disease Who Previously Completed Study MON-2021-001
Status:
RECRUITING
Status Verified Date:
2024-10
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 study is a multicenter 12-month open label extension study following Phase 1 Study MON-2021-001 with a single dose of monepantel MPL once daily QD for the treatment of individuals with MND
Detailed Description:
ALSMND is a progressive fatal neurodegenerative disease characterized by motor neuron loss resulting in muscle weakness and atrophy disability and eventually death from failure of the ventilatory muscles The median age of onset is 55 years and average survival is 3-5 years after onset of the first symptoms The only FDA-approved disease modifying medications confer only a modest survival benefit Given the poor prognosis and dearth of effective treatments clinical studies are of primary importance for people with ALSMND
Abnormal protein accumulation within motor neurons of the brain associates with the cause of ALSMND Inhibition of the mTOR signaling pathway slows disease progression in certain preclinical models of ALSMND and is suggested to provide synergy with the ALSMND standard-of-care drug riluzole PharmAust has shown that MPL and its major metabolite MPL sulfone MPLS have activity against mTOR signaling pathways in humans based on published data the inhibition of mTOR may be relevant to the treatment of ALSMND
This Phase I Open Label Extension will further test the hypotheses that MPL administration to individuals living with ALSMND will safely reduce disease associated protein accumulation in motor neurons and provide therapeutic benefit The safety and tolerability of oral monepantel administration and markers of efficacy will continue to be tested in the same participants that completed the Phase I Study MON-2021-001
A daily dose of 10 mgkg monepantel QD will be studied in the Open Label Extension Study MON-2023-001 to further evaluate long-term safety and efficacy in participants with MNDALS that completed the Phase I Study MON-2021-001 Based on the previous pre-clinical efficacy data and clinical safety data a dose of 10 mgkg QD is estimated to produce the most robust mTOR inhibition while still being well tolerated The 10 mgkg QD dose was the maximum dose evaluated in the Phase 1 Study
Abnormal protein accumulation within motor neurons of the brain associates with the cause of ALSMND Inhibition of the mTOR signaling pathway slows disease progression in certain preclinical models of ALSMND and is suggested to provide synergy with the ALSMND standard-of-care drug riluzole PharmAust has shown that MPL and its major metabolite MPL sulfone MPLS have activity against mTOR signaling pathways in humans based on published data the inhibition of mTOR may be relevant to the treatment of ALSMND
This Phase I Open Label Extension will further test the hypotheses that MPL administration to individuals living with ALSMND will safely reduce disease associated protein accumulation in motor neurons and provide therapeutic benefit The safety and tolerability of oral monepantel administration and markers of efficacy will continue to be tested in the same participants that completed the Phase I Study MON-2021-001
A daily dose of 10 mgkg monepantel QD will be studied in the Open Label Extension Study MON-2023-001 to further evaluate long-term safety and efficacy in participants with MNDALS that completed the Phase I Study MON-2021-001 Based on the previous pre-clinical efficacy data and clinical safety data a dose of 10 mgkg QD is estimated to produce the most robust mTOR inhibition while still being well tolerated The 10 mgkg QD dose was the maximum dose evaluated in the Phase 1 Study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?:
None