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Trial registered on ANZCTR


Registration number
ACTRN12622001025729
Ethics application status
Not required
Date submitted
18/07/2022
Date registered
22/07/2022
Date last updated
8/01/2024
Date data sharing statement initially provided
22/07/2022
Type of registration
Prospectively registered

Titles & IDs
Public title
Systemic Exposure Exploratory Open-Label Study of EmtinB to Determine Presence in Cerebrospinal Fluid (CSF)
Scientific title
A Phase 1, Single-Arm, Exploratory Open-Label Study Investigating Safety, Tolerability, and Pharmacokinetics of Subcutaneously Administered EmtinB in Healthy Adult Volunteers
Secondary ID [1] 307613 0
NSB1528-782-101
Universal Trial Number (UTN)
Trial acronym
Linked study record
This study is linked to the parent study ACTRN12622001019796

Health condition
Health condition(s) or problem(s) studied:
Neurodegenerative disorders 327087 0
Condition category
Condition code
Neurological 324240 324240 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Experimental: Single dose of EmtinB at planned dose level of 150 mg administered on a single occasion. EmtinB will be administered subcutaneously to the abdomen as a single dose in the clinic by trained, qualified personnel.
Intervention code [1] 324063 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 332059 0
Concentration of EmtinB in a CSF sample.
Timepoint [1] 332059 0
Participants will have a single sample collected at either 3 hours or 8 hours post-dose. This will be undertaken in a non-randomised fashion such that the first 4 participants have their sample collected at 3 hours post-dose and the last 4 have their sample collected at 8 hours post-dose.
Secondary outcome [1] 412024 0
Safety of EmtinB assessed by occurrence of treatment emergent adverse events recorded through observation and non-leading questioning in accordance with the Common Terminology Criteria for Adverse Events (CTCAE5.0) as well as through specific assessments to objectively assess for clinically significant changes from baseline in: - Laboratory evaluations (blood and urine samples for chemistry, coagulation studies, haematology and urinalysis); - Vital signs (temperature using an electronic probe, manually counted respiratory rate and pulse rate and blood pressure measured using a non-invasive automated blood pressure monitor); - Electrocardiograms; - Physical examinations.
Timepoint [1] 412024 0
Safety will be assessed at each point of contact from pre-dose on Day 1 until the final contact on Day 8 which will be 7 days post-dose.
Secondary outcome [2] 412025 0
Tolerability of EmtinB will be assessed through review of local injection site reactions, which will be assessed using:
- Local Injection Site Toxicity Grading Scale
- Visual Analogue Scale (VAS) for pain
- Photography of the injection site.
Timepoint [2] 412025 0
Tolerability will be assessed at each point of contact from immediately post-dose on Day 1 until the final contact on Day 8 which will be 7 days post-dose.
Secondary outcome [3] 412026 0
PK of EmtinB on plasma samples to assess Cmax, tmax,AUC, t1/2, Kel, CL/F, Vz/f and Css.
Timepoint [3] 412026 0
Blood samples collected at pre-dose, 0.5, 1, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 30, 36and 48 hours post-dose

Eligibility
Key inclusion criteria
1. Generally healthy with the exception of those medical conditions allowed per the inclusion/exclusion criteria.
2. Adult male and female participants aged greater than or equal to 18 and less than or equal to 60 years at the time of screening.
Minimum age
18 Years
Maximum age
60 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. Presence or history of any cardiovascular, gastrointestinal, renal, hepatic, respiratory, hematological,lymphatic, immunological, dermatological, neurological, musculoskeletal, connective tissue, genitourinary,endocrine or psychiatric diseases or disorders, which are determined as clinically relevant by the investigator as they would make implementation of the protocol or interpretation of the study data difficult, or would put the participant at risk by participation in the study in the opinion of the investigator..
2. Any evidence or treatment of malignancy (other than localized basal cell cancer, squamous cell skin cancer, or cancer in situ that has been resected or successfully treated with topical therapy e.g. Aldara) within the previous 5 years.
3. Abnormal ECG, and deemed clinically significant by the investigator, at screening and/or Day -1 defined as; QTcF >450 msec (males) or >470 msec (females).
4. Clinically significant renal disease, nephrectomy, renal transplant or estimated glomerular filtration rate of <90 mL/min/1.73 m^2 at screening based on Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation 2009.
5. Abnormal vital sign findings (after resting semi-supine for 5 minutes) defined as:
• Blood pressure that is >140 mm Hg or <90mm Hg systolic, or >90 mm Hg or <40 mm Hg diastolic at screening or Day -1.
• Pulse rate that is <40 or >100 bpm at screening or Day -1.
6. Used or are using over-the-counter medications, dietary/nutritional supplements (except for occasional paracetamol, up to 2 g in any 1 day) within 7 days prior to first dose on Day 1 until completion of the EOS visit.
7. Used or are using prescription medications (except stable female contraceptives that must continue uninterrupted for the duration of the study) within 14 days or 5 half-lives prior to first dose on Day 1 (whichever is longer) until completion of the EOS visit.
8. Used or are using systemic steroids (orally or intravenously administered) within 28 days prior to first dose on Day 1 until completion of EOS visit.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Withdrawn
Reason for early stopping/withdrawal
Other reasons/comments
Other reasons
Strategic decision of the company to withdraw from development in this indication
Date of first participant enrolment
Anticipated
23/01/2024
Actual
Date of last participant enrolment
Anticipated
23/01/2024
Actual
Date of last data collection
Anticipated
30/01/2024
Actual
Sample size
Target
8
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 25372 0
Nucleus Network - Melbourne
Recruitment hospital [2] 25373 0
Nucleus Network - Geelong
Recruitment postcode(s) [1] 41101 0
3004 - Melbourne
Recruitment postcode(s) [2] 41102 0
3220 - Geelong

Funding & Sponsors
Funding source category [1] 311880 0
Commercial sector/Industry
Name [1] 311880 0
NeuroScientific Biopharmaceuticals Ltd
Country [1] 311880 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
NeuroScientific Biopharmaceuticals Ltd
Address
Suite 5, 85 Forrest Street, Cottesloe WA 6011
Country
Australia
Secondary sponsor category [1] 313358 0
None
Name [1] 313358 0
Address [1] 313358 0
Country [1] 313358 0
Other collaborator category [1] 282369 0
Commercial sector/Industry
Name [1] 282369 0
Nucleus Network Pty Ltd
Address [1] 282369 0
Level 5, Burnet Tower, 89 Commercial Road, Melbourne, Victoria 3004
Country [1] 282369 0
Australia
Other collaborator category [2] 282796 0
Commercial sector/Industry
Name [2] 282796 0
Nucleus Network Pty Ltd
Address [2] 282796 0
235 Ryrie Street, Geelong, Victoria 3220
Country [2] 282796 0
Australia

Ethics approval
Ethics application status
Not required

Summary
Brief summary
This Phase 1 study will comprise a single group conducted in up to 8 participants who will receive a single dose of EmtinB in an open-label fashion. In addition to safety, tolerability and pharmacokinetics (PK), a single cerebrospinal fluid (CSF) sample will be collected from each participant via lumbar puncture to assess the ability of EmtinB to cross the blood brain barrier.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 120682 0
Dr Philip Ryan
Address 120682 0
Nucleus Network Pty Ltd, Level 5, Burnet Tower, 89 Commercial Road, Melbourne, Victoria 3004
Country 120682 0
Australia
Phone 120682 0
+61 3 8593 9800
Fax 120682 0
Email 120682 0
[email protected]
Contact person for public queries
Name 120683 0
Nucleus Network Melbourne
Address 120683 0
Level 5, Burnet Tower, 89 Commercial Road, Melbourne, Victoria 3004
Country 120683 0
Australia
Phone 120683 0
+61 1800 243 733
Fax 120683 0
Email 120683 0
[email protected]
Contact person for scientific queries
Name 120684 0
Simon Scott
Address 120684 0
NeuroScientific Biopharmaceuticals Ltd
Suite 5, 85 Forrest Street
Cottesloe 6011 WA
Country 120684 0
Australia
Phone 120684 0
+61 8 6382 1805
Fax 120684 0
Email 120684 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.