Clinical study with Talineuren for the treatment of Parkinson's disease.
Summary description of the study
NEON is a clinical study aimed at investigating the safety of Talineuren in patients with Parkinson's disease at a study center in the canton of Bern. Typically, patients with Parkinson's are treated with dopamine/levodopa medications to counteract the dopamine deficiency caused by the disease. In our research project, we want to find out whether the additional therapy with Talineuren is well tolerated and whether an effect can be measured. Talineuren contains the active ingredient GM1 (Monosialotetrahexosylganglioside), which is already on the market in other countries and is administered either intramuscularly or intravenously. InnoMedica produces GM1 in a new formulation called Talineuren, in which the active ingredient is encapsulated in very small lipid spheres at the nanometer scale. The active ingredient GM1 has shown good tolerability in other applications and has properties that protect nerve cells. Affected patients should be able to benefit as soon as possible. In patients with Parkinson's, the naturally occurring GM1 in the brain is reduced, which is why we conduct the NEON study in diseased individuals and not in healthy ones. Thus, in addition to collecting data on the safety and tolerability of Talineuren, data relevant for the efficacy assessment can already be collected. With this clinical study, we want to investigate in a first phase, where the dose is continuously increased (3 patients, so-called dose escalation), and in a second phase where a well-tolerated dose is administered multiple times (9 patients, so-called dose consolidation), how well tolerated Talineuren is and whether the first effects of efficacy can already be measured.
(BASEC)
Intervention under investigation
Part 1 of the study: gradual dose increase (dose escalation) in 3 patients until the maximum tolerable dose is reached or until intolerable side effects occur. The first dose is set very low at 6 mg of Talineuren. The increase occurs to 12 mg and 60 mg. From there, dose increments of 60 mg are made, up to a maximum of 720 mg. The maximum duration of Talineuren administration is 14 weeks.
Part 2 of the study: repeated administration of the same dose (dose consolidation) in 9 patients with the maximum tolerable dose determined in part 1 for 8 weeks.
In both parts, study participants receive Talineuren once a week as an infusion lasting about 1 hour.
Optional further treatment 1: patients from dose escalation have the opportunity for an 8-week further treatment with the study medication (720 mg/week).
Optional further treatment 2: all patients have the opportunity for a 16-week further treatment with the study medication (720 mg/week).
Optional further treatment 3:
- All previously enrolled study patients have the opportunity for an 8-month further treatment with the study medication (720 mg/week).
- 10 new patients will be enrolled in the study and treated for 8 months with the study medication (720 mg/week).
Optional further treatment 4:
- Study patients from parts I and II have the opportunity for a 4-month further treatment with the study medication (720 mg/week).
Optional further treatment 5:
- Study patients from parts I and II have the opportunity for a 12-month further treatment with the study medication (720 mg/week).
(BASEC)
Disease under investigation
Confirmed Parkinson's disease according to British Brain Bank criteria, disease stage according to Hoehn and Yahr 0 – 2.5, with Parkinson's medications.
(BASEC)
• Confirmed Parkinson's disease according to British Brain Bank criteria • Disease stage according to Hoehn and Yahr 0 – 2.5 with medications • No dementia according to MoCA (Montreal Cognitive Assessment) (BASEC)
Exclusion criteria
• Contraindication for this class of medications or known hypersensitivity or allergy • Atypical Parkinson-like syndrome or secondary parkinsonism • Comorbidities that could adversely affect the course of the study (BASEC)
Trial sites
Bern
(BASEC)
Sponsor
InnoMedica Schweiz AG
(BASEC)
Contact
Contact Person Switzerland
InnoMedica
+41 31 311 04 27
trials@clutterinnomedica.comInnoMedica
(BASEC)
Scientific Information
not available
Name of the authorising ethics committee (for multicentre studies, only the lead committee)
Ethics Committee Bern
(BASEC)
Date of authorisation
11.10.2021
(BASEC)
ICTRP Trial ID
NCT04976127 (ICTRP)
Official title (approved by ethics committee)
Safety evaluation of intravenous Talineuren (TLN) in patients with Parkinson’s disease (BASEC)
Academic title
Safety Evaluation of Intravenous Talineuren (TLN) in Parkinson's Disease-affected Patients (ICTRP)
Public title
Safety Evaluation of Intravenous Talineuren (TLN) in Parkinson's Disease-affected Patients (ICTRP)
Disease under investigation
Parkinson Disease (ICTRP)
Intervention under investigation
Drug: Talineuren (ICTRP)
Type of trial
Interventional (ICTRP)
Trial design
Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label). (ICTRP)
Inclusion/Exclusion criteria
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. (ICTRP)
not available
Primary and secondary end points
Occurence of adverse events (safety);Occurence of serious adverse events (safety);Occurence of other safety-related signs (safety) (ICTRP)
Levodopa challenge (LDC) test;Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS);Epworth Sleepiness Scale (ESS);Parkinson's Disease Questionnaire (PDQ-39);Change in Parkinson's medication;Starkstein Apathy Scale (SAS);Montreal Cognitive Assessment (MoCA);Beck's Depression Inventory (BDI);Non-Motor Symptoms Questionnaire (NMSQuest);Maximum Observed Drug Concentration (Cmax) in serum;Time of Maximum Drug Concentration (Tmax) in serum;Area Under the Curve to infinity (AUCinf.) in serum;half-life (t1/2);Clearance (CL);Volume of distribution (Vd) (ICTRP)
Registration date
not available
Incorporation of the first participant
not available
Secondary sponsors
not available
Additional contacts
not available
Secondary trial IDs
TLN/PD/1 (ICTRP)
Results-Individual Participant Data (IPD)
not available
Further information on the trial
https://clinicaltrials.gov/study/NCT04976127 (ICTRP)
Results of the trial
Results summary
not available
Link to the results in the primary register
not available