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Trial registered on ANZCTR


Registration number
ACTRN12625000189426
Ethics application status
Approved
Date submitted
30/09/2024
Date registered
18/02/2025
Date last updated
18/02/2025
Date data sharing statement initially provided
18/02/2025
Type of registration
Retrospectively registered

Titles & IDs
Public title
A Single-arm, Open-label, Single-dose, Phase I Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Recombinant Human Thrombopoietin for Injection (rhTPO) in Healthy Caucasian Volunteers
Scientific title
A Single-arm, Open-label, Single-dose, Phase I Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Recombinant Human Thrombopoietin for Injection (rhTPO) in Healthy Caucasian Volunteers
Secondary ID [1] 313076 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Thrombocytopenia 335313 0
Condition category
Condition code
Blood 331890 331890 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is a single-center, single-arm, open-label, single-dose phase I clinical study to evaluate pharmacokinetics, pharmacodynamics, safety and tolerability of Recombinant Human Thrombopoietin for Injection (rhTPO) in healthy Caucasian participants. Approximately 22 healthy Caucasian participants will be enrolled for this study. One dose level is planned in this study, and participants will receive a single abdominal subcutaneous injection at a dose of 300U/kg after entering the study.
Injection site assessment (ISR) will be performed at pre-dose, 0.5h, 1h±10min, 3h±15min, 6h±15min, 9h±15min, 12h±15min post-dose.
Participants need to fast for at least 10 hours prior to dosing and 4 hours after dosing.
Intervention code [1] 329633 0
Treatment: Drugs
Comparator / control treatment
No control group.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 339501 0
Serum rhTPO concentration
Timepoint [1] 339501 0
pre-dosing and 14 days after dosing
Primary outcome [2] 339502 0
Time to Peak Drug Concentration (Tmax)
Timepoint [2] 339502 0
pre-dosing and 14 days after dosing
Primary outcome [3] 339503 0
Peak Concentration (Cmax)
Timepoint [3] 339503 0
pre-dosing and 14 days after dosing
Secondary outcome [1] 440209 0
Incidence and severity of Adverse Event (AE), this will be assessed as a composite outcome
Timepoint [1] 440209 0
daily from signing informed consent form to 29 days after last dosing
Secondary outcome [2] 440210 0
Incidence and severity of Serious Adverse Event (SAE), this will be assessed as a composite outcome
Timepoint [2] 440210 0
daily from signing informed consent form to 29 days after last dosing
Secondary outcome [3] 440211 0
Incidence and severity of abnormal vital sign, this will be assessed as a composite outcome
Timepoint [3] 440211 0
daily from signing informed consent form to 29 days after last dosing

Eligibility
Key inclusion criteria
1. Healthy Caucasian adult male and female participants aged 18-50 years (inclusive) at the time of informed consent. Caucasian is defined as of European, Middle Eastern, or North African descent.
2. Male participants weighing no less than 50 kg. Female participants should not weigh less than 45 kg.
3. Body mass index (BMI) within the range 18.0 to 32.0 kg/m2 (inclusive).
Note: BMI = weight [kg] / (height [m])2
4. Participants are in good general health as determined by the investigator, based on a medical evaluation including medical history, vital signs, physical examination, 12-lead electrocardiogram, clinical safety laboratory tests;
Note: Any results outside of the reference range at screening or baseline may be repeated once per the Investigator’s discretion for the purpose of further determining eligibility.
5. Platelet counts must be within the normal range; measurements can repeat once for verification, if necessary.
6. Women of childbearing potential (WOCBP, see section 5.3.3) must have a negative serum or urine pregnancy test prior to the start of study drug and must not be breastfeeding, lactating or planning pregnancy during the study period. WOCBP who are not exclusively in same-sex relationships and males with partners of child-bearing potential must agree to use adequate contraception from signing the informed consent until 3 months after completion of the study. In addition, females must not donate ova (eggs) and males must not donate sperm from signing the informed consent until 3 months after completion of the study;
7. Volunteer to participate in this study, be able to understand and comply with the clinical study protocol requirements, and sign the informed consent form in writing.
Minimum age
18 Years
Maximum age
50 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1 History of hypersensitivity to components of study drug or analogs; history of anaphylaxis or other significant allergy in opinion of the investigators.
2 Known allergy to heparin or history of heparin-induced thrombocytopenia;
3 Received a platelet transfusion within 2 weeks or platelet-boosting medication within 4 weeks prior to dosing;
4 History of hospitalization or surgical procedure within 3 months prior to dosing, or bleeding from trauma or internal bleeding greater than 100 mL, or donating more than 100 mL of blood;
5 History or presence of autoimmune disease, malignancy, thromboembolic disease, bleeding disorders, cardiovascular, hepatic, renal, muscular, hematopoietic, respiratory, neurological, or psychiatric disorders capable of significantly altering the absorption, metabolism, or elimination of drugs, constituting a risk to the participant when taking the study drug; or interfering with the interpretation of data judged by the investigator;
Note: Uncomplicated cholecystectomy and appendectomy are permitted; History of fully resolved childhood asthma is not permitted.
6 History or presence of chronic diseases such as hypertension and diabetes (including type 1 and type 2 diabetes);
7 History of malignancy with the exception of successfully treated basal cell or squamous cell skin cancer with no evidence of recurrence for 5 years.
8 History of platelet clumping that prevents reliable measurement of platelet counts;
9 Positive for Hepatitis B surface antigen (HBsAg), Hepatitis C Virus (HCV) antibody and Human Immunodeficiency Virus (HIV) antibody, Syphilis antibody;
Note: Participants with positive hepatitis C antibody can be enrolled if a confirmatory negative hepatitis C RNA test is obtained.
10 Use of prescription medication within 14 days prior to dosing and 7 days prior to dosing for over the counter medication/vitamins/herbal supplements (with the exception of contraception, occasional paracetamol < 2g/day, and standard dose of multivitamins).
11 Consuption of alcohol, caffeinated products (coffee, tea, cola, chocolate), grapefruit fruits, or products containing grapefruit within 48 hours prior to dosing;
12 Consuption of any poppy seeds (e.g., orange and poppy seed muffin, poppy seed bread) within 48 hours prior to screening and admission.
13 History of drug abuse within 2 years prior to dosing; or positive alcohol/urine drug screen prior to dosing; Regular alcohol consumption within 6 months prior to dosing, i.e., on average more than 14 units of alcohol per week (1 unit = 360mL of beer or 45mL of spirits at 40% alcohol by volume or 150mL of wine);
14 Use of tobacco or nicotine products equivalent to more than 2 cigarettes a day on average within 3 months prior to dosing;
15 Have special dietary requirements and are unable to comply with the diet offered and the corresponding regulations;
16 Pregnant or breastfeeding female, or positive pregnancy test;
17 Individuals who cannot tolerate venipuncture or who suffer from needle and bloodsickness;
18 Participation in another clinical study of investigational drug within 3 months prior to dosing;
19 Other participants judged by the investigator to be unsuitable for participation.

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
Pharmacokinetics / pharmacodynamics
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
3/10/2024
Date of last participant enrolment
Anticipated
Actual
18/11/2024
Date of last data collection
Anticipated
Actual
17/12/2024
Sample size
Target
22
Accrual to date
Final
22
Recruitment in Australia
Recruitment state(s)
VIC

Funding & Sponsors
Funding source category [1] 317517 0
Commercial sector/Industry
Name [1] 317517 0
3SBIO AU PTY LTD
Country [1] 317517 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
3SBIO AU PTY LTD
Address
Country
Australia
Secondary sponsor category [1] 319820 0
None
Name [1] 319820 0
Address [1] 319820 0
Country [1] 319820 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 316231 0
Bellberry Human Research Ethics Committee A
Ethics committee address [1] 316231 0
https://bellberry.com.au/
Ethics committee country [1] 316231 0
Australia
Date submitted for ethics approval [1] 316231 0
14/08/2024
Approval date [1] 316231 0
17/09/2024
Ethics approval number [1] 316231 0

Summary
Brief summary
This study is testing the safety, tolerability (if any side effects occur), pharmacokinetics (PK; the amount of investigational drug or any breakdown products in your blood), pharmacodynamics (PD; how the investigational drug affects your body), and Immunogenicity (the ability of cells/tissues to provoke an immune response) of a single dose of an investigational drug called Recombinant Human Thrombopoietin for Injection- (rhTPO). This study drug has been marketed in China and other nine (9) countries since 2005, treating patients with primary immune thrombocytopenia (ITP) and chemotherapy-induced thrombocytopenia (CIT), under the trade name of TPIAO.
Participants will undergo screening, admission (baseline), administration and follow-up observation period. Participants will sign an informed consent form before any study procedures are performed. During screening, all participants will be screened for study eligibility within 28 days prior to administration. Eligible participants will be admitted to the clinical study ward no later than one day prior to dosing (D-1). Participants need to fast for at least 10 hours prior to dosing and 4 hours after dosing. Single abdominal subcutaneous injection of the study drug will be given on D1. The administration and follow-up observation period will be 29 days (D1~D29). Participants may be discharged at D7 at the judgment of the investigator. PK blood samples will be collected from D-1 to D14, tolerance and safety will be observed from D1 to D29, and blood samples for PD and ADA will be collected from screening to D29. After completion of relevant assessments on D29, participants will be considered as having completed this study.This is a single-center, single-arm, open-label, single-dose phase I clinical study to evaluate pharmacokinetics, pharmacodynamics, safety and tolerability of Recombinant Human Thrombopoietin for Injection (rhTPO) in healthy Caucasian participants. Approximately 22 healthy Caucasian participants will be enrolled for this study.
One dose level is planned in this study, and participants will receive a single abdominal subcutaneous injection at a dose of 300U/kg after entering the study.
Trial website
Trial related presentations / publications
Public notes
Subjects should have immunization with a COVID-19 vaccine 14 days prior to dosing or planned vaccination within 30 days after dosing and should be negative for COVID-19 based on the nasopharyngeal or oropharyngeal swab with the method of real-time reverse transcription-polymerase chain reaction (RT-PCR) at Screening and Day-1.

Contacts
Principal investigator
Name 137226 0
Dr Arockiaa Philo Aarthy Joseph
Address 137226 0
Nucleus Network Pty Ltd, Level 5, Burnet Tower, 89 Commercial Rd, Melbourne, Victoria,?3004 and Level 3, 549 St Kilda Road, Melbourne, Victoria, 3004
Country 137226 0
Australia
Phone 137226 0
+61 459 361 568
Fax 137226 0
Email 137226 0
[email protected]
Contact person for public queries
Name 137227 0
Yan Lu
Address 137227 0
3SBIO AU PTY LTD, Unit 26,9 Salisbury Road,Castle Hill NSW 2154 Australia
Country 137227 0
Australia
Phone 137227 0
+61280466801
Fax 137227 0
Email 137227 0
[email protected]
Contact person for scientific queries
Name 137228 0
Yan Lu
Address 137228 0
3SBIO AU PTY LTD, Unit 26,9 Salisbury Road,Castle Hill NSW 2154 Australia
Country 137228 0
Australia
Phone 137228 0
+61280466801
Fax 137228 0
Email 137228 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.