|E.1 Medical condition or disease under investigation
|Medical condition(s) being investigated
|Medical condition in easily understood language
|Diseases [C] - Nervous System Diseases [C10]
|Condition being studied is a rare disease
|E.2 Objective of the trial
|Main objective of the trial
|The study is an exploratory pilot study. Thus no formal sample size calculation or hypothesis testing will be done.
Main objective is to investigate the efficacy of 28 days NBMI treatment on motor and non-motor symptoms and heath related quality of life in patients with Progressive Supranuclear Palsy or Multiple Systems Atrophy disease.
| Študija je exploratorna pilotska študija.
Evaluirati učinkovitost zdravljenja z NBMI na motorične in nemotorične simtope ter z zdravjem povezano kakovost življenja bolnikov s progresivno supranuklearno paralizo (PSP) ali multiplo sistemsko atrofijo (MSA)
|Secondary objectives of the trial
|To investigate the safety and tolerability of 28 days oral administration of NBMI in patients with PSP or MSA.
To investigate the efficacy of NBMI daily oral administration for 28 days on fatigue in MSA and PSP patients.
To investigate the efficacy of NBMI daily oral administration for 28 days on depression in MSA and PSP patients.
|Trial contains a sub-study
|Full title, date and version of each sub-study and their related objectives
|FDG PET imaging substudy.
Objective is to explore changes from baseline in brain metabolism as evaluated with FDG- PET- CT brain imaging, compared to placebo treatment Pharmacokinetic parameters derived from plasma concentration of NBMI in patients with PSP or MSA.
|Principal inclusion criteria
|1. Patient has clinically confirmed diagnosis of PSP or MSA according to the current criteria.
2. Patient has a brain MRI finding consistent with diagnosis of PSP or MSA at screening
3. Patient is aged 50 years to 85 years inclusive at screening age.
4. Patient is on stable therapy for PSP or MSA for at least 1 month prior to screening visit.
5. If patient received i.v amantadine treatment, the last infusion must have been administered at least 6 months prior to the screening.
|Principal exclusion criteria
|1. Known hystory or presence of clinically significant other neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, genitourinary, psychiatric or cardiovascular disease or other condition which in opinion of investigators would jeapardise the safety of the subject or impact the validity of study results.
2. Known or suspected hypesensitivity or idiosyncratic reaction to NBMI or any other drug substance with similar activity
3. Patient has known contraindication for MRI imaging
|E.5 End points
|Primary end point(s)
|Changes from baseline in motor and non-motor symptoms and heath related quality of life in patients with Progressive supranuclear palsy or Multiple Ssystems atrophy disease compared to placebo treatment.
|Timepoint(s) of evaluation of this end point
| Mean change in PSPRS and FAB individual scales scores from Baseline - V1 to V2 (D29) and V4 (D57) in PSP patients. and mean change in UMSARS
and NMSS individual scales scores Baseline - V1 to V2 (D29) and V4 (D57) in MSA patients
Changes in QOL individual scores from baseline by MSA questionnaire in MSA patients and in EQ-5D score in PSP patients compared to placebo
treatment - V1 to V2 (D29) and V4 (D57)
|Secondary end point(s)
|Adverse events in terms of frequency and severity compared to placebo treatment
Changes from baseline in fatigue as measured by PFS in MSA and PSP patients compared to placebo treatment ,
Changes from baseline in depression as measured by Beck’s Depression Inventory in MSA patients and GDS in PSP patients compared to placebo treatment .
|Timepoint(s) of evaluation of this end point
|Adverse events in terms of frequency and severity compared to placebo treatment - during the whole study.
Changes Mean change in PFS score from Baseline - V1 to V2 (D29) and V1 to V4 (D57) in PSP and MSA patient.s compared to placebo treatment
Changes in BDI scale (MSA patients) and GDS scale (PSP patients) score from Baseline - V1 to V2 (D29) and to V4 (D57) compared to placebo
|E.6 and E.7 Scope of the trial
|Scope of the trial
|Other scope of the trial description
|Raziskovalne končne točke.
|Trial type and phase
|Human pharmacology (Phase I)
|First administration to humans
|Other trial type description
|Therapeutic exploratory (Phase II)
|Therapeutic confirmatory (Phase III)
|Therapeutic use (Phase IV)
|E.8 Design of the trial
| Comparator of controlled trial
|Other medicinal product(s)
|Number of treatment arms in the trial
The trial involves single site in the Member State concerned
| The trial involves multiple sites in the Member State concerned
|The trial involves multiple Member States
|E.8.6 Trial involving sites outside the EEA
|Trial being conducted both within and outside the EEA
|Trial being conducted completely outside of the EEA
|Trial has a data monitoring committee
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|E.8.9 Initial estimate of the duration of the trial
|In the Member State concerned years
|In the Member State concerned months
|In the Member State concerned days