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

NCT06431971 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: LIBRA (TLN/PD/2)
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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 II/III trial.

Conditions

Parkinson Disease

Outcome Measures

Primary Outcomes (1)

• Movement Disorder Society Unified Parkinson's Disease Rating Scale score

  The Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) will be used by the patient and physician to evaluate various symptoms of Parkinson disease including non-motor and motor experiences of daily living and motor complications. The MDS-UPDRS Scale consists of 4 parts: * Part 1 (Nonmotor aspects of experiences of daily living) with 13 items. * Part 2 (Motor aspects of experiences of daily living) with 13 items. * Part 3 (Motor examination) with 18 items. * Part 4 (Motor complications) with 6 items. Each item is rated with 0=normal, 1=slight, 2=mild, 3=moderate, 4=severe. The lower the score, the fewer / less severe the symptoms.

  Baseline, week 7, 11, 15, 19 and 22

Secondary Outcomes (3)

• Parkinson disease Quality of Life Questionnaire score

  Baseline, week 19

• Montreal Cognitive Assessment score

  baseline, week 19

• Change in Levodopa equivalent daily dose

  Through study completion, an average of 22 weeks

Other Outcomes (3)

• Number of IMP-related adverse events

  From the date of the first administration until week 22

• Proportion of feasibility parameters

  Up to week 22

• Mean values of Biomarkers

  Baseline, week 19

Study Arms (2)

Talineuren

EXPERIMENTAL

Participants receive standard of care PD treatment + 720 mg of Talineuren i.v. weekly for 18 infusions (18 weeks).

Drug: Talineuren

Placebo

PLACEBO COMPARATOR

Participants receive standard of care PD treatment + placebo (0.9% NaCl) i.v. weekly for 18 infusions (18 weeks).

Drug: Placebo

Interventions

Talineuren DRUG

Talineuren infusion weekly

Also known as: TLN, Liposomal GM1

Talineuren

Placebo DRUG

Placebo infusion weekly

Also known as: 0.9% NaCl

Placebo

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.
• Male and female subjects, aged 30 to 85 years.
• Confirmed PD according to British brain bank criteria.
• Hoehn and Yahr Stage 0 - 2.5 on medication.
• Stable dopaminergic PD treatment (including DBS) for a month at least.
• Absence of dementia confirmed by cognitive testing (MoCA ≥24).

You may not qualify if:

• Previous treatment with Talineuren (i.e. participants from NEON trial are not allowed)
• Contraindications to the class of drugs under study, e.g., known hypersensitivity or allergy to class of drugs or the investigational products.
• Women who are pregnant or breast feeding, or planning to become pregnant during the course of the trial or in the 12 weeks following the trial.
• Lack of safe contraception, defined as:
• Female participants of childbearing potential, not willing to use double method of contraception (hormonal and mechanical) for the entire study duration.
• Note: Female participants who are surgically sterilised / hysterectomised or post-menopausal for longer than 2 years are not considered as being of child bearing potential.
• Male participants, not using and not willing to use a medically reliable method of contraception for the entire study duration, such as condoms or who are not using any other method considered sufficiently reliable by the investigator in individual cases.
• Other clinically significant concomitant diseases states (e.g., renal failure, hepatic dysfunction, cardiovascular disease etc.) that is are not under stable control.
• Known or suspected non-compliance, drug or alcohol abuse.
• Inability to follow the procedures of the trial, e.g., due to language problems, psychological disorders etc. of the participant.
• Participation in another trial with an investigational drug within the 30 days preceding and during the present trial.
• Enrolment of the investigator, his/her family members, employees and other dependent persons.
• Subject has an atypical parkinsonian syndrome or secondary parkinsonism.
• Patients with comorbidity that may interfere with the course of the trial.
• Patients who are not considered to be eligible to participate in clinical trial by the investigator.

Sponsors & Collaborators

• InnoMedica Schweiz AG: lead

Study Sites (1)

Neurologisches Institut Konolfingen

Konolfingen, 3510, Switzerland

Location

MeSH Terms

Conditions

Parkinson Disease

Interventions

Saline Solution

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2024
• First Posted: May 29, 2024
• Study Start: August 1, 2024
• Primary Completion: November 1, 2025
• Study Completion: December 1, 2025
• Last Updated: May 29, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

CH (1)