Ignite Creation Date:
2024-05-06 @ 4:09 PM
Last Modification Date:
2024-10-26 @ 2:05 PM
Study NCT ID:
NCT04894240
Status:
COMPLETED
Last Update Posted:
2023-12-21
First Post:
2021-05-05
Brief Title:
A Study of Monepantel in Individuals With Motor Neurone Disease
Sponsor:
PharmAust Ltd
Organization:
PharmAust Ltd
Study Overview
Official Title:
A Phase I Tolerability Safety Pharmacokinetics and Preliminary Efficacy Study of Oral Monepantel in Individuals With Motor Neurone Disease
Status:
COMPLETED
Status Verified Date:
2023-12
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:
Amyotrophic lateral sclerosis Motor Neurone Disease ALSMND is a rare and invariably fatal neurological disease ALSMND has a terribly high burden on patients family and carers and carries great socioeconomic burden Current best treatment options are expensive and attempt to control disease progression and manage symptoms while offering no cure Better treatments are wanting
Monepantel is a well-known veterinary drug registered as a livestock wormicide in 39 countries The industry collaborator PharmAust Ltd has found that monepantel shows off-target activity inhibiting a cellular signaling system controlled by mammalian target of rapamycin mTOR This stops cancer growth and reduces protein accumulation in diseased cells PharmAust has already tested monepantel in humans and pet dogs in Phase I and II anti-cancer clinical trials respectively in Australia Data from these trials show that monepantel treatment associates with an exceptionally high safety profile mTOR signaling inhibition and anticancer activity
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 An alternative mTOR inhibitor rapamycin is currently the subject of an ALSMND clinical trial in humans investigating control of disease progression Monepantel has a different structure to rapamycin and an apparently better safety profile
This Phase I Clinical Trial hypothesis is that monepantel administration to individuals living with ALSMND will safely reduce disease associated protein accumulation in motor neurons and provide therapeutic benefit To test this hypothesis the safety and tolerability of oral monepantel administration and markers of efficacy will be tested in individuals living with ALSMND in a dose escalating Phase III Clinical Trial To mitigate risk only patients with sporadic and certain known familial types of ALS will be eligible To further mitigate risk the monepantel starting dose will be reduced a calculated five-fold compared to that already used in human cancer patients and already demonstrated to be safe and effective as an mTOR inhibitor Dependent upon incremental outcomes three higher doses may then be tested each for minimally 28 days with a duration at the optimal dose of at least six months
Monepantel is a well-known veterinary drug registered as a livestock wormicide in 39 countries The industry collaborator PharmAust Ltd has found that monepantel shows off-target activity inhibiting a cellular signaling system controlled by mammalian target of rapamycin mTOR This stops cancer growth and reduces protein accumulation in diseased cells PharmAust has already tested monepantel in humans and pet dogs in Phase I and II anti-cancer clinical trials respectively in Australia Data from these trials show that monepantel treatment associates with an exceptionally high safety profile mTOR signaling inhibition and anticancer activity
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 An alternative mTOR inhibitor rapamycin is currently the subject of an ALSMND clinical trial in humans investigating control of disease progression Monepantel has a different structure to rapamycin and an apparently better safety profile
This Phase I Clinical Trial hypothesis is that monepantel administration to individuals living with ALSMND will safely reduce disease associated protein accumulation in motor neurons and provide therapeutic benefit To test this hypothesis the safety and tolerability of oral monepantel administration and markers of efficacy will be tested in individuals living with ALSMND in a dose escalating Phase III Clinical Trial To mitigate risk only patients with sporadic and certain known familial types of ALS will be eligible To further mitigate risk the monepantel starting dose will be reduced a calculated five-fold compared to that already used in human cancer patients and already demonstrated to be safe and effective as an mTOR inhibitor Dependent upon incremental outcomes three higher doses may then be tested each for minimally 28 days with a duration at the optimal dose of at least six months
Detailed Description:
None
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