PSP or MSA
Conditions
Interventions
Product Name: NBMI
Product Code: NBMI
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: emeramide
CAS Number: 100-00-5
Current Sponsor code: NBMI
Other descriptive name: N1,N3-BIS(2-MERCAPTOETHY
Sponsors
NBMI Science Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 10 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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. ;Secondary Objective: 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. ;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
| Measure | Time frame |
|---|---|
| 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 treatment ). | — |
Countries
Slovenia
Contacts
Public ContactTanja Turk
CRS d.o.o.
Outcome results
None listed