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

NCT04976127 | Terminated Phase 1 DMC

• 1 other identifier: TLN/PD/1
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Parkinson Disease

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (15)

• Levodopa challenge (LDC) test

  8 to 134 weeks

• Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)

  8 to 134 weeks

• Epworth Sleepiness Scale (ESS)

  8 to 134 weeks

• Parkinson's Disease Questionnaire (PDQ-39)

  8 to 134 weeks

• Change in Parkinson's medication

  8 to 134 weeks

Study Arms (3)

Talineuren dose escalation

EXPERIMENTAL

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 repeated dose

EXPERIMENTAL

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 dose consolidation with intrapatient dosing (additional patients)

EXPERIMENTAL

8 months repeated doses of 720mg GM1 Ganglioside (Amendment 3).

Drug: Talineuren

Interventions

Talineuren DRUG

Talineuren is a liposomal formulation of the GM1 Ganglioside for intravenous administration

Also known as: liposomal GM1

Talineuren dose consolidation with intrapatient dosing (additional patients); Talineuren dose escalation; Talineuren repeated dose

Eligibility Criteria

• Age: 30 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• InnoMedica Schweiz AG: lead

Study Sites (1)

Neurologisches Institut Konolfingen

Konolfingen, Canton of Bern, 3510, Switzerland

Location

Related Publications (2)

• 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.

  PMID: 40359409 DERIVED

• 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.

  PMID: 37534999 DERIVED

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Part 1: dose escalation phase, 3 patients with Parkinson's disease, (2 cohorts as in 1+2 patients, sequential inclusion between the first and 2nd patient) Part 2: repeated dose administration phase, 9 patients with Parkinson's disease (1 cohort) Part 3: Dose consolidation with intrapatient dosing, 10 patients with Parkinson's disease (1 cohort)

Study Record Dates

• First Submitted: May 16, 2021
• First Posted: July 26, 2021
• Study Start: December 11, 2021
• Primary Completion: August 6, 2025
• Study Completion: August 6, 2025
• Last Updated: April 22, 2026

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CH (1)