Clinical Trials Register

Summary
EudraCT Number:	2018-000506-34
Sponsor's Protocol Code Number:	EMERA006
National Competent Authority:	Slovenia - JAZMP
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2018-03-09
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Slovenia - JAZMP

A.2

EudraCT number

2018-000506-34

A.3

Full title of the trial

EMERA006- A pilot, exploratory, randomised, placebo-controlled, double blinded, cross-over, Phase 2a study to explore efficacy and safety of NBMI treatment in patients with Progressive Supranuclear Palsy (PSP) or Multiple System Atrophy (MSA).

EMERA006 -Pilotno, exploratorno, randomizirano, dvojno slepo, s placebom kontrolirano navzkrižno klinično preskušanje faze 2 a zdravljenja za oceno učinkovitosti in varnosti NBMI pri bolnikih s PSP ali MSA.

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

EMERA006 - A pilot, exploratory, randomised, placebo-controlled, double blinded, cross-over, Phase 2a study to explore efficacy and safety of NBMI treatment in patients with Progressive Supranuclear Palsy (PSP) or Multiple System Atrophy (MSA).

A.3.2

Name or abbreviated title of the trial where available

EMERA006

A.4.1

Sponsor's protocol code number

EMERA006

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	NBMI Science Ltd.
B.1.3.4	Country	Ireland
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	NBMI Science Ltd
B.4.2	Country	Ireland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	CRS d.o.o.
B.5.2	Functional name of contact point	Tanja Turk
B.5.3	Address:
B.5.3.1	Street Address	Ukmarjeva ulica 6
B.5.3.2	Town/ city	Ljubljana
B.5.3.3	Post code	1000
B.5.3.4	Country	Slovenia
B.5.4	Telephone number	+38615442501
B.5.5	Fax number	+38615442502
B.5.6	E-mail	tanja.turk@crs.si

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	Mercury toxicity
D.3 Description of the IMP
D.3.1	Product name	NBMI
D.3.2	Product code	NBMI
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	emeramide
D.3.9.1	CAS number	100-00-5
D.3.9.2	Current sponsor code	NBMI
D.3.9.3	Other descriptive name	N1,N3-BIS(2-MERCAPTOETHYL) ISOPHTHALAMIDE
D.3.9.4	EV Substance Code	SUB184226
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	300
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Capsule, hard
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

PSP or MSA

PSP ali MSA

E.1.1.1

Medical condition in easily understood language

PSP or MSA

PSP ali MSA

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

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)

E.2.2

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.

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

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.

E.3

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.

E.4

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

E.5.1

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.

E.5.1.1

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)

E.5.2

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 .

E.5.2.1

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 ).

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Exploratory endopoints.

Raziskovalne končne točke.

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

E.8.1.6

Cross over

Yes

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LSLV

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Participation in Phase II b trial will be considered.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2019-02-04

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-04-18

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2021-06-30

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials