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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04787887




Registration number
NCT04787887
Ethics application status
Date submitted
4/03/2021
Date registered
9/03/2021
Date last updated
5/04/2021

Titles & IDs
Public title
A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy Volunteers
Scientific title
A Randomized, Double-blind, 2-treatment, 2-period, Crossover Phase I Study to Compare the PK, Safety and Tolerability of 60 IU/kg of Abcertin, and EU-sourced Cerezyme® in Healthy Volunteers Following a Single Intravenous Administration
Secondary ID [1] 0 0
ACTRN12619001399189p
Secondary ID [2] 0 0
ISU302-005
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Gaucher Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Abcertin
Treatment: Drugs - EU-sourced Cerezyme

Active Comparator: Abcertin - Abcertin 60IU/kg

Active Comparator: Cerezyme - EU-sourced Cerezyme


Treatment: Drugs: Abcertin
60IU/kg Single Intravenous Administration

Treatment: Drugs: EU-sourced Cerezyme
60IU/kg Single Intravenous Administration
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
AUC0-inf
Timepoint [1] 0 0
Predose(0 minute), during infusion(10, 20, 40, 60, 90, 120 minutes), and after infusion( 5, 10, 20, 30, 40, 50, 60 ,70, 80, 90, 120 minutes)
Secondary outcome [1] 0 0
Cmax
Timepoint [1] 0 0
Predose(0 minute), during infusion(10, 20, 40, 60, 90, 120 minutes), and after infusion( 5, 10, 20, 30, 40, 50, 60 ,70, 80, 90, 120 minutes)
Secondary outcome [2] 0 0
tmax
Timepoint [2] 0 0
Predose(0 minute), during infusion(10, 20, 40, 60, 90, 120 minutes), and after infusion( 5, 10, 20, 30, 40, 50, 60 ,70, 80, 90, 120 minutes)
Secondary outcome [3] 0 0
AUC0-last
Timepoint [3] 0 0
Predose(0 minute), during infusion(10, 20, 40, 60, 90, 120 minutes), and after infusion( 5, 10, 20, 30, 40, 50, 60 ,70, 80, 90, 120 minutes)
Secondary outcome [4] 0 0
t½
Timepoint [4] 0 0
Predose(0 minute), during infusion(10, 20, 40, 60, 90, 120 minutes), and after infusion( 5, 10, 20, 30, 40, 50, 60 ,70, 80, 90, 120 minutes)
Secondary outcome [5] 0 0
CL
Timepoint [5] 0 0
Predose(0 minute), during infusion(10, 20, 40, 60, 90, 120 minutes), and after infusion( 5, 10, 20, 30, 40, 50, 60 ,70, 80, 90, 120 minutes)
Secondary outcome [6] 0 0
Vz
Timepoint [6] 0 0
Predose(0 minute), during infusion(10, 20, 40, 60, 90, 120 minutes), and after infusion( 5, 10, 20, 30, 40, 50, 60 ,70, 80, 90, 120 minutes)

Eligibility
Key inclusion criteria
1. Subject must have been able to give voluntary written informed consent prior to any
study-related procedures.

2. Subject must have been available for the entire study period.

3. Subject was male or female aged between = 18 and = 45 years old.

4. Subject had a body mass index (BMI) between = 18.50 and = 30.00 kg/m2 and weighed
between 55 and 105 kg, inclusive.

5. Female subject of childbearing potential must have been non-pregnant and non-lactating
and must have had a negative pregnancy test at Screening and at each admission to the
clinical research center.

6. Female subject of childbearing potential, with a fertile male sexual partner, must
have used adequate contraception from Screening until 90 days after the Follow-up
Visit. Adequate contraception is identified as using hormonal contraceptives or an
intrauterine device combined with at least one of the following forms of
contraception: a diaphragm or cervical cap, or a condom.

7. Male subject must have used adequate contraception and must not have donated sperm
from first admission to the clinical research center until 90 days after the Follow-up
Visit. Adequate contraception for the male subject and his female partner is defined
as using hormonal contraceptives or an intrauterine device combined with at least one
of the following forms of contraception: a diaphragm or cervical cap, or a condom.

8. Subject must have been healthy, as determined by the Principal Investigator, based on
medical history, physical examination, 12-lead ECG and laboratory evaluations
(hematology, blood chemistry, coagulation and urinalysis tests).

9. All values for the subject's clinical laboratory tests of blood and urine should not
have been clinically significant, as judged by the Principal Investigator.
Minimum age
18 Years
Maximum age
45 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. Intake of any investigational drug in another study within 30 days (or 5 half-lives,
whichever was greater) prior to the intake of the IP in this study or had received the
last dose of IP more than 30 days prior (or 5 half-lives, whichever was greater) but
who were on extended follow-up, or planned intake of an investigational drug (other
than for this study) during the course of this study.

2. With ongoing symptoms that had indicated acute diseases within 28 days prior to IP
administration; acute disease referred to any new onset of symptoms/signs or diagnosis
of disease, whether infectious, inflammatory, traumatic, etc., in origin (regardless
of whether or not the subject was hospitalized).

3. With any medical history that might have affected IP distribution, metabolism and
excretion (e.g., hepatic or renal disease).

4. Positive screen on hepatitis B surface antigen (HbsAg), hepatitis C virus (HCV)
antibodies or anti-human immunodeficiency virus (HIV) type 1 and type 2 antibodies. If
a potential subject was considered by the Investigator to have false positive result
(e.g., HIV antibodies) at Screening, a repeat test should have been done as soon as
possible and if the retest was negative, the subject could have been considered
eligible for the study.

5. Had clinically significant hypersensitivity or severe allergic reactions (either
spontaneous or following IP administration), also including known or suspected
clinically relevant drug hypersensitivity to any components of the IP or comparable
drugs, including Latex.

6. Had vaccination within 3 months prior to the first IP administration or planned a
vaccination before the Follow-up Visit.

7. Safety laboratory tests with the following results:

1. Aspartate aminotransferase (AST, also known as serum glutamic-oxaloacetic
transaminase) or alanine aminotransferase (ALT, also known as serum
glutamic-pyruvic transaminase) > 1.5 times the upper limit of normal (ULN), or

2. Total bilirubin (TBL) > 1.5 times ULN.

8. Subject who had immune deficiency or medication with immunosuppressive agents.

9. Treatment with any medication, prescribed or over-the-counter (OTC) products including
herbal remedies, within 14 days prior to Day 1 or longer if the medication had a long
half-life, unless agreed as not clinically significant by the Investigator and
Sponsor. Exceptions: hormonal contraceptives, acetaminophen = 3 g/day, vitamins at
daily recommended doses.

10. Had donated whole blood products (e.g., plasma, platelets) within 60 days, or
transfused within 20 days before Screening.

11. History of alcohol or drug abuse or drug addiction (including cannabis products)
within the last 12 months before Screening.

12. Subject was willing to comply with the alcohol restrictions. The subject should have
refrained from drinking any alcohol within 72 hours prior to Day -1 and should not
have consumed more than 3 - 4 units of alcohol per day, to a maximum of 14 units of
alcohol per week (1 unit of 10 mL of pure alcohol is equal to 12 ounces [360 mL] of
beer, 5 ounces [150 mL] of wine or 1.5 ounces [45 mL] of 80-proof distilled spirits)
throughout the study.

13. Heavy smoker (> 5 cigarettes/day) or the subject could not stop smoking during the
study period while inpatient at the clinical research center.

14. Positive tests for drugs of abuse or alcohol at Screening or Day -1 (admission to the
clinical research center).

15. Family member or employee of the Investigator or clinical research center staff or
study team.

16. Those who were not suitable for participation in the study based on the Investigator's
judgement, for any reason including laboratory test results.

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)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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
Crossover
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/01/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/10/2020
Sample size
Target
Accrual to date
Final
42
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Scientia Clinical Research Limited - Randwick
Recruitment postcode(s) [1] 0 0
2031 - Randwick

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
ISU Abxis Co., Ltd.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Primary Objective:

To compare the pharmacokinetics of Abcertin to the reference product, EU-sourced Cerezyme,
after single intravenous administration of 60 IU/kg.

Secondary Objective:

To compare the safety, tolerability and immunogenicity of Abcertin to the reference
formulation, EU-sourced Cerezyme, after single intravenous administration of 60 IU/kg.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04787887
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Charlotte Lemech, MD
Address 0 0
Scientia Clinical Research Limited
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04787887