Gaucher DiseasePhase 1NCT04787887
A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy Volunteers
A Phase 1 study testing Abcertin and EU-sourced Cerezyme in people with Gaucher Disease, run by ISU Abxis Co., Ltd. at 1 site in Australia.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 45 yearsHealthy volunteers can take part
- Conditions the study is for
- Gaucher Disease
- What is being tested?
- Abcertin and EU-sourced Cerezyme
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 1 siteAustralia
- Study dates
- 29 Jan 2020to 26 Oct 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Subject must have been able to give voluntary written informed consent prior to any study-related procedures.
- Subject must have been available for the entire study period.
- Subject was male or female aged between ≥ 18 and ≤ 45 years old.
- Subject had a body mass index (BMI) between ≥ 18.50 and ≤ 30.00 kg/m2 and weighed between 55 and 105 kg, inclusive.
- 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.
- 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.
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- 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.
- 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).
- 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.
Who cannot join
- 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.
- 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).
- With any medical history that might have affected IP distribution, metabolism and excretion (e.g., hepatic or renal disease).
- 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.
- 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.
- Had vaccination within 3 months prior to the first IP administration or planned a vaccination before the Follow-up Visit.
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- Safety laboratory tests with the following results:
- 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
- Total bilirubin (TBL) > 1.5 times ULN.
- Subject who had immune deficiency or medication with immunosuppressive agents.
- 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.
- Had donated whole blood products (e.g., plasma, platelets) within 60 days, or transfused within 20 days before Screening.
- History of alcohol or drug abuse or drug addiction (including cannabis products) within the last 12 months before Screening.
- 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.
- Heavy smoker (> 5 cigarettes/day) or the subject could not stop smoking during the study period while inpatient at the clinical research center.
- Positive tests for drugs of abuse or alcohol at Screening or Day -1 (admission to the clinical research center).
- Family member or employee of the Investigator or clinical research center staff or study team.
- Those who were not suitable for participation in the study based on the Investigator's judgement, for any reason including laboratory test results.
Trial locations and contacts
1 site in 1 country.
Australia1 site
Scientia Clinical Research Limited
Randwick, New South Wales, 2031
About this study
Official 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
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.
Read the detailed description
This is a phase 1, single-center, randomized, double-blind, two-way crossover study employing Abcertin and EU-sourced Cerezyme in healthy volunteers between the ages of 18 and 45 years (inclusive). The study aimed to evaluate the PK, safety, tolerability and immunogenicity of Abcertin compared with EU-sourced Cerezyme when administered as a single dose.
- Conditions
- Gaucher Disease
- Being tested
- Abcertin, EU-sourced Cerezyme
- Keywords
- Gaucher Disease, ISU ABXIS Co., Ltd., ISU302
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AbcertinDrug
Also called Imiglucerase
60IU/kg Single Intravenous Administration
EU-sourced CerezymeDrug
Also called Imiglucerase
60IU/kg Single Intravenous Administration
| Study group | Role | What they receive |
|---|---|---|
| Abcertin | Active comparator | Abcertin 60IU/kg Receives: Drug: Abcertin; Drug: EU-sourced Cerezyme |
| Cerezyme | Active comparator | EU-sourced Cerezyme Receives: Drug: Abcertin; Drug: EU-sourced Cerezyme |
What is being measured
The primary outcome is the main question the study is designed to answer.
AUC0-inf
Area under the concentration-time curve (AUC) from time zero to time infinity
Measured: 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 outcomes (6)
Cmax
Maximum plasma concentration
Measured: 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)
tmax
Time to Cmax
Measured: 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)
AUC0-last
AUC from time zero to the time of the last measurable plasma concentration
Measured: 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)
t½
Terminal half-life
Measured: 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)
CL
Total body clearance
Measured: 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)
Vz
Volume of distribution based on the terminal phase
Measured: 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)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2021
- Study start29 Jan 2020
- Primary completion27 Sep 2020
- Study completion26 Oct 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- ISU Abxis Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Charlotte Lemech, MD, Principal investigator, Scientia Clinical Research Limited
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04787887
- Sponsor's protocol ID
- ISU302-005
- Other IDs
- ACTRN12619001399189p
- Status last verified
- Apr 2021
- First submitted
- 4 Mar 2021
- Last updated
- 5 Apr 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT04787887
- Acronym / sponsor ID
- ISU302-005
- Conditions
- Gaucher Disease
- Keywords
- Gaucher Disease, ISU ABXIS Co., Ltd., ISU302
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- ISU Abxis Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 29 Jan 2020 (actual)
- Primary completion
- 27 Sep 2020 (actual)
- Study completion
- 26 Oct 2020 (actual)
- First posted
- 9 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a phase 1, single-center, randomized, double-blind, two-way crossover study employing Abcertin and EU-sourced Cerezyme in healthy volunteers between the ages of 18 and 45 years (inclusive). The study aimed to evaluate the PK, safety, tolerability and immunogenicity of Abcertin compared with EU-sourced Cerezyme when administered as a single dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Abcertin | Active comparator | Drug: Abcertin, Drug: EU-sourced CerezymeAbcertin 60IU/kg |
| Cerezyme | Active comparator | Drug: Abcertin, Drug: EU-sourced CerezymeEU-sourced Cerezyme |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- Subject must have been able to give voluntary written informed consent prior to any study-related procedures.
- Subject must have been available for the entire study period.
- Subject was male or female aged between ≥ 18 and ≤ 45 years old.
- Subject had a body mass index (BMI) between ≥ 18.50 and ≤ 30.00 kg/m2 and weighed between 55 and 105 kg, inclusive.
- 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.
Key exclusion criteria (18 listed)
- 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.
- 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).
- With any medical history that might have affected IP distribution, metabolism and excretion (e.g., hepatic or renal disease).
- 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.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AbcertinAlso: Imiglucerase60IU/kg Single Intravenous Administration | Drug | Abcertin, Cerezyme |
| EU-sourced CerezymeAlso: Imiglucerase60IU/kg Single Intravenous Administration | Drug | Abcertin, Cerezyme |
Primary outcome
AUC0-inf
Time frame: 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)
Area under the concentration-time curve (AUC) from time zero to time infinity
6 secondary outcomes
Cmax
Time frame: 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)
tmax
Time frame: 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)
AUC0-last
Time frame: 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)
t½
Time frame: 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)
CL
Time frame: 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)
Vz
Time frame: 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)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Apr 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Charlotte Lemech, MD principal investigator
Scientia Clinical Research Limited
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Abcertin and EU-sourced Cerezyme in people with Gaucher Disease, run by ISU Abxis Co., Ltd. at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
AUC0-inf (time frame: 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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Apr 2021; AzTrials last refreshed its copy on 26 Oct 2026.