Safety Evaluation of Intravenous Talineuren (TLN) in Parkinson's Disease-affected Patients (NEON)

This study is an open-label, single ascending dose escalation followed by a multiple administration dose at the maximal suitable dose (MSD). The investigational Medicinal Product (IMP) is given as an add-on therapy.

Talineuren consists of GM1 (monosialotetrahexosylganglioside), the pharmacologically active ingredient, associated with a proprietary lipid formulation assembled as liposomes.

The primary objective is to demonstrate the safety of TLN administration intravenously in Parkinson patients.

Secondary objectives are the determination of the maximal suitable dose based on the safety profile and preliminary efficacy, as well as the determination of the pharmacokinetics (PK) profile.

Study Overview

• Status: Terminated
• Conditions: Parkinson Disease
• Intervention / Treatment: Drug: Talineuren

Detailed Description

The ganglioside lipid GM1 has been described in the literature as a neuroprotective agent.

Several clinical studies have shown that GM1 improves the condition of Parkinson's disease patients.

Talineuren consists of the pharmacologically active ingredient GM1, associated with a proprietary lipid formulation assembled as liposomes. Talineuren has been developed to improve the delivery and bioavailability of GM1.

The primary objective of this trial is to demonstrate the feasibility and safety of intravenous Talineuren administration in Parkinson's disease patients.

The secondary objectives are:

• The determination of the recommended phase 2 dose based on the safety profile and preliminary efficacy.
• The determination of the pharmacokinetics (PK) profile.

This trial aims to investigate the safety of the novel formulation of GM1, Talineuren.

To that extent a three-part trial was designed: Part 1- Dose escalation Part 2- Dose consolidation Part 3- Dose consolidation with intrapatient dosing

Part 1- rapid dose escalation scheme from 6 mg to 720 mg of Talineuren formulated GM1 in 3 patients. Optional treatment prolongations for 8 weeks (Amendment 1), 16 weeks (Amendment 2), 8 months (Amendment 3), 4 months (Amendment 4), and 12 months (Amendment 5).

Part 2- multiple dosing of Talineuren over 8 weeks in 9 patients to validate the safety profile of the maximum suitable dose. Optional treatment prolongations for 16 weeks (Amendment 2), 8 months (Amendment 3), 4 months (Amendment 4), and 12 months (Amendment 5).

Part 3- rapid dose escalation scheme from 6 mg to 720 mg of Talineuren followed by multiple doses of 720mg Talineuren for up to 8 months in 10 patients (Amendment 3). Additional Follow-up visit (washout timepoint) 4 months after final assessment (Amendment 6).

• Study Type: Interventional
• Enrollment (Actual): 22
• Phase: Phase 1

Contacts and Locations

Study Locations

• Switzerland
  • Canton of Bern
    • Konolfingen, Canton of Bern, Switzerland, 3510
      • Neurologisches Institut Konolfingen

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 30 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Informed consent as documented by signature.
• Confirmed Parkinson's disease according to British brain bank criteria.
• Hoehn and Yahr Stage 0 - 2.5 on medication.
• Stable on PD treatment for a month at least.
• Absence of dementia confirmed by cognitive testing (MoCA >25).

Exclusion Criteria:

• Contraindications to the class of drugs under study, e.g., known hypersensitivity or allergy to class of drugs or the investigational product.
• Women who are pregnant or breast feeding, or planning to become pregnant during the course of the trial or in the 3 months following the trial.
• Lack of safe contraception in women with childbearing potential
• Other clinically significant concomitant disease states (e.g., renal failure, hepatic dysfunction, cardiovascular disease, etc) that is not under stable control.
• Subject has an atypical parkinsonian syndrome or secondary parkinsonism.
• Patients with comorbidity that may interfere with the course of the trial.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Talineuren dose escalation; 14 doses of GM1 Ganglioside 6, 12, 60, 120, 180, 240, 300, 360, 420, 480, 540, 600, 660, 720 mg. Optional treatment prolongations for 16 weeks (Amendment 2), 8 months (Amendment 3), 4 months (Amendment 4) and 12 months (Amendment 5).	Drug: Talineuren; Talineuren is a liposomal formulation of the GM1 Ganglioside for intravenous administration; Other Names: liposomal GM1
Experimental: Talineuren repeated dose; 8 repeated doses of GM1 Ganglioside tbd from the escalation dose (maximum suitable dose). Optional treatment prolongations for 16 weeks (Amendment 2), 8 months (Amendment 3), 4 months (Amendment 4) and 12 months (Amendment 5).	Drug: Talineuren; Talineuren is a liposomal formulation of the GM1 Ganglioside for intravenous administration; Other Names: liposomal GM1
Experimental: Talineuren dose consolidation with intrapatient dosing (additional patients); 8 months repeated doses of 720mg GM1 Ganglioside (Amendment 3).	Drug: Talineuren; Talineuren is a liposomal formulation of the GM1 Ganglioside for intravenous administration; Other Names: liposomal GM1

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurence of adverse events (safety)	Number and kinds of adverse events (AEs)	8 to 134 weeks
Occurence of serious adverse events (safety)	Number and kinds of serious adverse events (SAEs)	8 to 134 weeks
Occurence of other safety-related signs (safety)	Number and kinds of other safety-related signs	8 to 134 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum Observed Drug Concentration (Cmax) in serum	Pharmacokinetics (PK) of total GM1 in serum over the first 96 h	8 to 15 weeks
Time of Maximum Drug Concentration (Tmax) in serum	Pharmacokinetics (PK) of total GM1 in serum over the first 96 h	8 to 15 weeks
Area Under the Curve to infinity (AUCinf.) in serum	Pharmacokinetics (PK) of total GM1 in serum over the first 96 h	8 to 15 weeks
half-life (t1/2)	Pharmacokinetics (PK) of total GM1 in serum over the first 96 h	8 to 15 weeks
Clearance (CL)	Pharmacokinetics (PK) of total GM1 in serum over the first 96 h	8 to 15 weeks
Volume of distribution (Vd)	Pharmacokinetics (PK) of total GM1 in serum over the first 96 h	8 to 15 weeks
Levodopa challenge (LDC) test	Assessing the patients' condition via the LDC test (MDS-UPDRS-3 score)	8 to 134 weeks
Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)	Assessing the patients' condition via the test	8 to 134 weeks
Epworth Sleepiness Scale (ESS)	Assessing the patients' condition via the test	8 to 134 weeks
Parkinson's Disease Questionnaire (PDQ-39)	Assessing the patients' condition via the test	8 to 134 weeks
Change in Parkinson's medication	Assessing the patients' condition via the change in their pre-existing Parkinson's medication	8 to 134 weeks
Starkstein Apathy Scale (SAS)	Assessing the patients' condition via the test	8 to 134 weeks
Montreal Cognitive Assessment (MoCA)	Assessing the patients' condition via the test	8 to 134 weeks
Beck's Depression Inventory (BDI)	Assessing the patients' condition via the test	8 to 134 weeks
Non-Motor Symptoms Questionnaire (NMSQuest)	Assessing the patients' condition via the test	8 to 134 weeks

Collaborators and Investigators

• Sponsor: InnoMedica Schweiz AG

Publications and helpful links

General Publications

• Halbherr S, Lerch S, Bellwald S, Polakova P, Bannert B, Roumet M, Charles RP, Walter MA, Bernasconi C, Halbherr VL, Peitsch C, Baumgartner PC, Kaufmann C, Aires V, Mattle HP, Kaelin-Lang A, Hartmann A, Schuepbach M. Safety and tolerability of intravenous liposomal GM1 in patients with Parkinson disease: A single-center open-label clinical phase I trial (NEON trial). PLoS Med. 2025 May 13;22(5):e1004472. doi: 10.1371/journal.pmed.1004472. eCollection 2025 May.
• Roy R, Paul R, Bhattacharya P, Borah A. Combating Dopaminergic Neurodegeneration in Parkinson's Disease through Nanovesicle Technology. ACS Chem Neurosci. 2023 Aug 16;14(16):2830-2848. doi: 10.1021/acschemneuro.3c00070. Epub 2023 Aug 3.

Study record dates

Study Major Dates

• Study Start (Actual): December 11, 2021
• Primary Completion (Actual): August 6, 2025
• Study Completion (Actual): August 6, 2025

Study Registration Dates

• First Submitted: May 16, 2021
• First Submitted That Met QC Criteria: July 14, 2021
• First Posted (Actual): July 26, 2021

Study Record Updates

• Last Update Posted (Actual): April 22, 2026
• Last Update Submitted That Met QC Criteria: April 17, 2026
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Synucleinopathies; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; Movement Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Parkinson Disease
• Other Study ID Numbers: TLN/PD/1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No