NCT06431971

Estimates of the Short-term Efficacy of Talineuren (TLN) and Placebo in Patients With Parkinson Disease

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
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Sponsor: InnoMedica Schweiz AG · Industry
• Sex: All
• Age: 30 Years – 85 Years
• Healthy volunteers: Not accepted

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 II/III 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 Alzheimer's 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 (i.e. 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 and/or 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 Parkinson's 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 II/III trial.

Conditions

• Parkinson Disease

Interventions

Type	Name	Description
DRUG	Talineuren	Talineuren infusion weekly
DRUG	Placebo	Placebo infusion weekly

Timeline

• Start date: 2024-08-01
• Primary completion: 2025-11-01
• Completion: 2025-12-01
• First posted: 2024-05-29
• Last updated: 2024-05-29

Locations

1 site across 1 country: Switzerland