Ignite Creation Date:
2024-06-16 @ 11:49 AM
Last Modification Date:
2024-10-26 @ 3:30 PM
Study NCT ID:
NCT06431971
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-05-29
First Post:
2024-05-16
Brief Title:
Estimates of the Short-term Efficacy of Talineuren TLN and Placebo in Patients With Parkinson Disease
Sponsor:
InnoMedica Schweiz AG
Organization:
InnoMedica Schweiz AG
Study Overview
Official Title:
Estimates of the Short-term Efficacy of Talineuren TLN and Placebo in Patients With Parkinson Disease A Randomized Placebo-controlled Double-blinded Parallel 2-arm Multi-centre Pilot Trial
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-05
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 double-blinded placebo controlled to estimate the short-term efficacy of Talineuren The investigational Medicinal Product IMP is administrated 18 times intravenously as an add-on therapy to the standard of care Parkinson medication
Talineuren is a liposomal formulation containing GM1 monosialotetrahexosylganglioside as the pharmacological active substance
The results of this pilot study are essential for the sample size calculation of a subsequent larger phase IIIII trial
Talineuren is a liposomal formulation containing GM1 monosialotetrahexosylganglioside as the pharmacological active substance
The results of this pilot study are essential for the sample size calculation of a subsequent larger phase IIIII trial
Detailed Description:
The ganglioside lipid GM1 monosialotetrahexosylganglioside has attracted attention in scientific literature as a promising neuroprotective agent Research suggests that GM1 ganglioside holds promise not only in the treatment of neurodegenerative disorders like Parkinson disease PD and Alzheimers disease but also in promoting nerve regeneration post-injury Furthermore investigations into its potential to improve cognitive function and memory underscore its versatility as a therapeutic agent Numerous clinical studies have demonstrated its therapeutic potential in treating PD patients
Talineuren TLN represents a novel approach to harnessing the therapeutic benefits of GM1 TLN is a liposomal formulation comprising GM1 as its pharmacologically active ingredient which is expected to cross the blood-brain barrier more efficiently as free GM1 and therefore is able to deliver more GM1 to the brain This innovative composition is designed to optimize the neuroprotective effects of GM1
Study Description
This study is designed as a double-blinded placebo-controlled trial to evaluate the short-term efficacy of TLN in PD management The investigational Medicinal Product IMP TLN is weekly intravenously administered 18 times as an add-on therapy alongside patients current standard-of-care PD medication Talineuren encapsulating GM1 within liposomes is anticipated to facilitate enhanced delivery and bioavailability of the neuroprotective agent GM1
Objectives
The primary objective is to obtain statistical estimates of change from baseline and variance for TLN and placebo and to compare these between groups for MDS-UPDRS part III score in the off medication state ie Levodopa challenge test LCT motor symptoms evaluated by physician
Secondary objectives are to obtain the change from baseline and variance of TLN and placebo and compare these between the groups for
MDS-UPDRS total score part I part II part III off part IV
MDS-UPDRS part I non-motor symptoms in daily life
MDS-UPDRS part II motor symptoms in daily life
MDS-UPDRS part III on medication
MDS-UPDRS total part IV motor complications
Proportion of patients meeting or exceeding the minimum clinically important difference MCID in motor and non-motor symptoms MDS-UPDRS and quality of life PDQ-39 over time
Quality of life PDQ-39
Mental condition MoCA
Parkinson medication LEDD
Research objectives biomarkers
-Assessment of literature-described biomarkers prognostic predictive monitoring andor response biomarkers pre- and post-TLN or placebo intervention
Through evaluation and statistical analysis this study seeks to elucidate the therapeutic potential of TLN in addressing the multifaceted challenges of Parkinsons disease By providing insights into treatment efficacy medication usage symptom management and quality of life improvements our findings aim to inform future advancements in PD management and enhance patient care
The results of this pilot study are essential for the sample size calculation of a subsequent larger phase IIIII trial
Talineuren TLN represents a novel approach to harnessing the therapeutic benefits of GM1 TLN is a liposomal formulation comprising GM1 as its pharmacologically active ingredient which is expected to cross the blood-brain barrier more efficiently as free GM1 and therefore is able to deliver more GM1 to the brain This innovative composition is designed to optimize the neuroprotective effects of GM1
Study Description
This study is designed as a double-blinded placebo-controlled trial to evaluate the short-term efficacy of TLN in PD management The investigational Medicinal Product IMP TLN is weekly intravenously administered 18 times as an add-on therapy alongside patients current standard-of-care PD medication Talineuren encapsulating GM1 within liposomes is anticipated to facilitate enhanced delivery and bioavailability of the neuroprotective agent GM1
Objectives
The primary objective is to obtain statistical estimates of change from baseline and variance for TLN and placebo and to compare these between groups for MDS-UPDRS part III score in the off medication state ie Levodopa challenge test LCT motor symptoms evaluated by physician
Secondary objectives are to obtain the change from baseline and variance of TLN and placebo and compare these between the groups for
MDS-UPDRS total score part I part II part III off part IV
MDS-UPDRS part I non-motor symptoms in daily life
MDS-UPDRS part II motor symptoms in daily life
MDS-UPDRS part III on medication
MDS-UPDRS total part IV motor complications
Proportion of patients meeting or exceeding the minimum clinically important difference MCID in motor and non-motor symptoms MDS-UPDRS and quality of life PDQ-39 over time
Quality of life PDQ-39
Mental condition MoCA
Parkinson medication LEDD
Research objectives biomarkers
-Assessment of literature-described biomarkers prognostic predictive monitoring andor response biomarkers pre- and post-TLN or placebo intervention
Through evaluation and statistical analysis this study seeks to elucidate the therapeutic potential of TLN in addressing the multifaceted challenges of Parkinsons disease By providing insights into treatment efficacy medication usage symptom management and quality of life improvements our findings aim to inform future advancements in PD management and enhance patient care
The results of this pilot study are essential for the sample size calculation of a subsequent larger phase IIIII trial
Study Oversight
Has Oversight DMC:
None
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?:
None