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Trial registered on ANZCTR


Registration number
ACTRN12620000572965
Ethics application status
Approved
Date submitted
19/03/2020
Date registered
15/05/2020
Date last updated
15/05/2020
Date data sharing statement initially provided
15/05/2020
Date results information initially provided
15/05/2020
Type of registration
Retrospectively registered

Titles & IDs
Public title
A Phase 1 Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of the AUM001 in Healthy Volunteers
Scientific title
A Phase 1, Randomized, Double-Blind, Placebo-Controlled Study
To Evaluate The Safety, Tolerability, And Pharmacokinetics Of Multiple Ascending Doses Of AUM001 In Normal Healthy Volunteers
Secondary ID [1] 300631 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Oncology; This is a healthy volunteer study. It is proposed that the drug will be evaluated for the treatment of patients with a range of solid and liquid tumours in Phase II. 316403 0
Condition category
Condition code
Cancer 314668 314668 0 0
Any cancer

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
AUM001 capsules will be administered in total 3 Cohorts with multiple ascending doses.
Subjects will receive active study drug AUM001 and matching placebo every other day over 13 days
Cohort 1: 5 mg (administered as 1 X 5 mg capsule)
Cohort 2: 10 mg (administered as 1 x 10 mg capsule)
Cohort 3: 20 mg (administered as 2 x 10 mg capsule)

IP will be administered via appropriate trained site staff in presence of medical personnel.
Each time an IMP is dispensed to a participant, received from the supplier and / or returned or destroyed, the occurrence will be documented on the accountability log for the purpose of adherence.
Subsequent Cohort starts only after the preceding cohort finishes. Each participant will only be enrolled in one cohort.
Intervention code [1] 316955 0
Treatment: Drugs
Comparator / control treatment
Matching placebo capsules administered per Cohort will be identical to the above cohort dose of investigational product.
The Placebo presentation consist of Microcrystalline Cellulose in White Opaque Hard Gellatine Capsules Size 2.
Control group
Placebo

Outcomes
Primary outcome [1] 322991 0
To evaluate the safety and tolerability of AUM001 following oral administration of multiple ascending doses ranging from 5 mg to 20 mg in healthy subjects.
Timepoint [1] 322991 0
Adverse events will be monitored throughout the study from the date of informed consent signed.
Vital signs will be measured at screening,on Day 1 pre-dose, and within 2 hours of dosing , and at at approximately 6, 12, 24 (Day 2), 48 (Day 3), 72 hours (Day 4) post-first dose, and every 24 hours thereafter (pre-dose on Days 5, 6, 7, 8, 9, 10, 11, and 12), and on Day 13: pre-dose and at approximately 1, 4, 8, 12, 16, 24 (Day 14), 48 (Day 15), and 72 (Day 16) hours post-last dose.
Triplicate ECG will be performed on pre-dose ECG only on day 1 and Single ECG recordings will be performed at approximately 1, 2, 4, 8, 12, 24 (Day 2), 48 (Day 3), 72 (Day 4) hours post-first dose, and every 24 hours thereafter before dosing (pre-dose on Days 5, 6, 7, 8, 9, 10, 11, 12, and 13). And before discharge on Day 14.
Continuous telemetry monitoring will be initiated pre dose on Day 1 and continue throughout the first 72 hours of the study
Hematology, biochemistry, coagulation, and urinalysis: pre-dose on Days -1 and on Days 2, 4, 6, 8, 10, and 12, and at discharge.
Coagulation will also be collected 2 hours post-first dose.
Urine pregnancy test and urine drug screen: before first dosing.
Secondary outcome [1] 380440 0
To characterize the pharmacokinetic (PK) profile of AUM001 following multiple oral doses in healthy subjects.

For Day 1, the following parameters will be calculated:

1) AUC0-48: area under the concentration-time curve from time zero to time 48 hours using the linear trapezoidal rule
2) Cmax: maximum observed concentration
3) Tmax: time of observed Cmax

For Day 13, the following parameters will be calculated:
1) AUC0-48(ss): area under the concentration-time curve for one dosing interval (t) at steady-state. In this study t = 48 hours .
2) Tmax ss: time of observed Cmax ss at steady-state
3) Cmax ss: maximum observed concentration at steady-state
4) Cmin ss: minimum observed concentration at steady-state
5) Fl(%): percentage of fluctuation
6) Cav(ss): average concentration at steady-state calculated from trapezoidal AUC data after multiple doses, as AUC0-t ss / t
7) AUC0-t(pred): predicted area under the concentration-time curve from time zero to the last non-zero concentration, after removal of the influence of the previous doses
8) AUC0-inf(pred): predicted area under the concentration-time curve from time zero to infinity (extrapolated)
9) T½ el: apparent elimination half-life
10) Kel: apparent elimination rate constant
11) CL/F(ss): total body clearance
Timepoint [1] 380440 0
A total of 26 PK blood samples will be collected:
Day 1: pre-dose and at 1, 2, 4, 8, 12, 16, 24, and 36 (Day 2) hours post-dose;
Day 3: pre-dose and 1 hour post- dose,
Day 9: pre-dose and 1 hour post-dose,
Day 11: pre-dose and 1 hour post-dose,
Day 13: pre-dose and at 1, 2, 4, 8, 12, 16, 24 (Day 14), 36 (Day 14), 48 (Day 15), and 72 (Day 16) hours post-last dose.
Pre-dose samples will be collected at approximately the same time each day and within 10 minutes prior to dosing.
Secondary outcome [2] 380441 0
To identify a safe dose range of AUM001 in preparation for patient studies. this is a secondary objective
Timepoint [2] 380441 0
Safety data including vital signs, ECG data as well as laboratory results and the AUM001 plasma concentration will be evaluated across all cohorts to decide to continue the prescribed dose level, to decrease the next dose level, to repeat a dose level or to not evaluate any additional dosage, based on consideration of the clinical significance of several safety, tolerability and PK parameters.

The blinded clinical and laboratory safety data as well as the blinded analysed Plasma PK data for all subjects within a cohort will be evaluated in aggregate once all subjects have completed the day 16 assessment

Eligibility
Key inclusion criteria
1) Male or female, light smokers, greater than or equal to 18 and less than or equal to 50 years of age, with BMI greater than 18.5 and less than 30.0 kg/m2 and body weight greater than or equal to 50.0 kg for males and less than or equal to 45.0 kg for females.
2) Female subjects must be non-pregnant, non-lactating, postmenopausal for at least 1 year, surgically sterile, or agree to use an acceptable form of contraception from the time of signing informed consent until 30 days after Study Completion. Females of childbearing potential who are sexually active with a non-sterile male partner (sterile male partners are defined as men vasectomized since at least 6 months) must be willing to use one of the following acceptable contraceptive:
3) Male subjects who are not vasectomized for at least 6 months, and who are sexually active with a female partner of childbearing potential must be willing to use one of the following acceptable contraceptive methods from the first study drug administration until at least 90 days after the last study drug administration:
4) Male subjects with a pregnant partner must agree to use a condom from the first study drug administration until at least 90 days after the last study drug administration.
5) Male subjects must be willing not to donate sperm until 90 days following the last study drug administration.
6) Subjects will be required to read, understand, and sign the informed consent and will be able to spend the days specified in the study schedule confined in a facility under study rules.
7) Subjects will have no history of cardiac disease.
8) Each subject’s baseline Safety Laboratory Assessment values must have no clinically significant abnormalities. This includes hematology, clinical chemistry, and urine analysis.
9) Subjects must be healthy volunteers with no active or chronic diseases/disorders,
10) Subjects will not have any allergy to the test article or to the constituents of the capsules
Minimum age
18 Years
Maximum age
50 Years
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1) Women who are nursing or pregnant.
2) Any surgical or medical condition that may interfere with the absorption, distribution, metabolism, or excretion of the test article.
3) Acute disease state
4) Admitted alcohol abuse or consumption of more than 2 standard units per day in the last 6 months.
5) Any clinically important deviation from normal limits in physical examination, vital signs, or clinical laboratory test results.
6) Positive serologic findings for human immunodeficiency virus (HIV) antibodies, hepatitis B surface antigen (HBsAg), and/or HCV antibodies.
7) Positive urine drug screen or alcohol test
8) History of allergy to any component of the test article.
9) Use of any prescription drug within 30 days prior to first dosing.
10) Use of any over-the-counter drugs including herbal supplements and Traditional Chinese Medicines within 14 days prior to first dosing.
11) Diets that alter metabolism
12) Participation in another clinical trial within 3 months prior to first dosing.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Participants will be randomly assigned to AUm001 dosing or placebo based on the randomization scheme. The pharmacist will dispense the study drug for individual subject dosing according to the randomization code.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Randomization scheme will be created to determine the treatment allocation of each subject enrolled
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint(s)
Pharmacokinetics
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 15955 0
CMAX Clinical Research Pty Ltd - Adelaide
Recruitment postcode(s) [1] 29441 0
5000 - Adelaide

Funding & Sponsors
Funding source category [1] 305054 0
Commercial sector/Industry
Name [1] 305054 0
AUM Biosciences Pty Ltd
Address [1] 305054 0
PO Box 545 Summer Hill NSW 2130
Country [1] 305054 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
AUM Biosciences Pty Ltd
Address
PO Box 545 Summer Hill NSW 2130
Country
Australia
Secondary sponsor category [1] 305421 0
None
Name [1] 305421 0
Address [1] 305421 0
Country [1] 305421 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 305442 0
Bellberry Limited
Ethics committee address [1] 305442 0
123 Glen Osmond Road Eastwood
South Australia 5063
Ethics committee country [1] 305442 0
Australia
Date submitted for ethics approval [1] 305442 0
04/09/2019
Approval date [1] 305442 0
24/10/2019
Ethics approval number [1] 305442 0

Summary
Brief summary
This Is a First In Human Phase 1 Single Center, Randomized Double-Blind, Placebo-Controlled Study To Evaluate The Safety Tolerability And Pharmacokinetics of MultipleE Ascending Doses of AUM001 In Normal Healthy Volunteers


This study will evaluate the safety, tolerability and pharmacokinetics of multiple ascending doses of AUM001 in healthy volunteers.

You may be eligible to join this study if you are aged 18 and above and below 50, are healthy with no active or chronic diseases/disorders

Participants in this study are randomly allocated (by chance) to one of two groups in one of three cohorts in ascending doses

Safety, tolerability and pharmacokinetics will be assessed at regular intervals using clinical examinations and blood tests.

This study is not open to cancer patients, and should not be promoted as such. Recruitment will be restricted to those volunteers identified as fitting the selection criteria by the Phase I Clinical trial Unit.

his is a healthy volunteer study. It is proposed that the drug will be evaluated for the treatment of patients with a range of solid and liquid tumours"
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 100402 0
Dr Sepehr Shakib
Address 100402 0
CMAX Clinical Research
Level 5, 18a North Terrace
Adelaide SA 5000
Country 100402 0
Australia
Phone 100402 0
+61 411 100 278
Fax 100402 0
Email 100402 0
Sepehr.Shakib@sa.gov.au
Contact person for public queries
Name 100403 0
Dr John Patava
Address 100403 0
AUM Biosciences Pty Ltd
PO Box 545 Summer Hill NSW 2130
Country 100403 0
Australia
Phone 100403 0
+61 498 071 249
Fax 100403 0
Email 100403 0
johnp@aumbiosciences.com
Contact person for scientific queries
Name 100404 0
Dr John Patava
Address 100404 0
AUM Biosciences Pty Ltd
58 Gipps St
Collingwood Vic 3066

Country 100404 0
Australia
Phone 100404 0
+61 498 071 249
Fax 100404 0
+61 498 071 249
Email 100404 0
harishd@aumbiosciences.com

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
As this is a Phase 1 study, only aggregate data may be posted/published.
What supporting documents are/will be available?
No other documents available
Summary results
Have study results been published in a peer-reviewed journal?
No
Other publications
Have study results been made publicly available in another format?
No
Results – basic reporting
Results – plain English summary
None