Hemophilia APhase 1NCT05251090
A Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII ，Fc Fusion Protein for Injection
A Phase 1 study testing ADVATE and FRSW107 in people with Hemophilia A, run by Hangzhou Gensciences Biopharmaceutical Co., Ltd. at 6 sites in China.
Trial summary
Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection (FRSW107) Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in Severe Hemophilia A. Read more about this study
- Who is it for?
- Ages Up to 12 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- ADVATE and FRSW107
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 6 sitesChina
- Study dates
- 16 Jun 2021to 9 May 2022
- 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 Up to 12 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- The activity of the coagulation factor VIII (FVIII:C) < 1%. Less than 6 years old Patients previously treated with FVIII concentrate (s) for a minimum of 50 exposure days (EDs) prior to study entry. 6 years old to 12 years old Patients previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry.
- Normal prothrombin time or INR < 1.3.
- Negative lupus anticoagulant.
Who cannot join
- Hypersensitive to any of the excipients of the test materials (e.g. allergic to murine or hamster origin heterologous proteins).
- History of hypersensitivity or anaphylaxis associated with any FVIII or II immunoglobulin administration.
- Current FVIII inhibitor-positive or history of FVIII inhibitor-positive.
- Other coagulation disorder(s) in addition to hemophilia A.
- Infusion of any products containing FVIII within 72 h prior to administration.
- Significant hepatic or renal impairment (ALT and AST > 2×ULN; serum bilirubin level > 2 × upper limit of normal (ULN), BUN > 2×ULN, Cr > 2.0 ULN).
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- One or more clinically significant tests for Human Immunodeficiency Virus (HIV), Antisyphilitic spirulina (TPHA) and Hepatitis C Virus (HCV) Antibody.
- Patients who received any anticoagulant or antiplatelet therapy within one week prior screening or need to receive an anticoagulant or antiplatelet therapy during the period of clinical trials.
- Patients having major surgery or receiving blood or bood components transfusion within 4 weeks prior screening or having planned major surgery schedule during the study.
- Patients who previously participated in the other clinical trials within one month prior to administration.
- Any life-threatening disease or condition which, according to the investigator's judgment, could not benefit from the trial participation.
- Patient who is considered by the other investigators not suitable for clinical study.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
China6 sites
Beijing Children's Hospital,Capital Medical University
Beijing, Beijing Municipality, 100045
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan, 610000
Nanfang Hospital of Southern Medical University
Guangzhou, Guangzhou, 510515
The Affiliated Hospital of Qingdao University
Qingdao, Shandong, 266000
Shenzhen Children's Hospita
Shenzhen, Guangdong, 518000
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
Wuhan, Hubei, 430000
About this study
Official title: A Phase I, Multicentre, Open-label Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection in Children With Severe Hemophilia A
Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection (FRSW107) Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in Severe Hemophilia A.
- Conditions
- Hemophilia A
- Being tested
- ADVATE, FRSW107
- Keywords
- Hemophilia A;Pharmacokinetics;Child;Safety and Tolerability
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ADVATEDrug
50 international units (IU)/kg, a single dose.
FRSW107Drug
Also called Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection
50 IU/kg, a single dose.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Subjects(up to 12 years of age) received two treatments: 50 IU/kg ADVATE in the first period, followed by 50 IU/kg FRSW107 in the second period. Receives: Drug: ADVATE; Drug: FRSW107 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum measured concentration of FVIII:C (Cmax)
Measured by aPTT Clotting Assay.
Measured: Pre-dose and post dose up to 8 days.
Time required for the concentration of the drug to reach half of its original value (T1/2)
Measured by aPTT Clotting Assay.
Measured: Pre-dose and post dose up to 8 days
Area Under the Curve to Infinity (AUC)
Measured by aPTT Clotting Assay.
Measured: Pre-dose and post dose up to 8 days.
The measure of the efficiency of the body to remove the drug and the unit is the volume of the plasma or blood cleared of drug per unit time (CL).
Measured by aPTT Clotting Assay.
Measured: Pre-dose and post dose up to 8 days.
Secondary outcomes (2)
Number of participants with treatment-related adverse events as assessed by CTCAE V5.0.
Adverse events related to Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection according to Common Terminology Criteria for Adverse Events (CTCAE) NCI.V5.0.
Measured: Post dose up to 32 days.
Development of Inhibitor
Measured by the Nijmegen-Modified Bethesda Assay.
Measured: Pre-dose and post dose up to 32 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Feb 2022
- Study start16 Jun 2021
- Primary completion15 Nov 2021
- Study completion9 May 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Runhui Wu, PhD, Principal investigator, Beijing Children's HospitalXiaoling Wang, MA.Sc, Principal investigator, Beijing Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05251090
- Sponsor's protocol ID
- CTR20220279
- Status last verified
- Aug 2023
- First submitted
- 11 Feb 2022
- Last updated
- 22 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Multicentre, Open-label Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection in Children With Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT05251090
- Acronym / sponsor ID
- CTR20220279
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A;Pharmacokinetics;Child;Safety and Tolerability
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 16 Jun 2021 (actual)
- Primary completion
- 15 Nov 2021 (actual)
- Study completion
- 9 May 2022 (actual)
- First posted
- 22 Feb 2022 (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 assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection (FRSW107) Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in Severe Hemophilia A.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Drug: ADVATE, Drug: FRSW107Subjects(up to 12 years of age) received two treatments: 50 IU/kg ADVATE in the first period, followed by 50 IU/kg FRSW107 in the second period. |
Who may qualify
- Ages
- Up to 12 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- The activity of the coagulation factor VIII (FVIII:C) < 1%. Less than 6 years old Patients previously treated with FVIII concentrate (s) for a minimum of 50 exposure days (EDs) prior to study entry. 6 years old to 12 years old Patients previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry.
- Normal prothrombin time or INR < 1.3.
- Negative lupus anticoagulant.
Key exclusion criteria (12 listed)
- Hypersensitive to any of the excipients of the test materials (e.g. allergic to murine or hamster origin heterologous proteins).
- History of hypersensitivity or anaphylaxis associated with any FVIII or II immunoglobulin administration.
- Current FVIII inhibitor-positive or history of FVIII inhibitor-positive.
- Other coagulation disorder(s) in addition to hemophilia A.
- Infusion of any products containing FVIII within 72 h prior to administration.
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 |
|---|---|---|
| ADVATE50 international units (IU)/kg, a single dose. | Drug | Arm 1 |
| FRSW107Also: Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection50 IU/kg, a single dose. | Drug | Arm 1 |
Primary outcome
Maximum measured concentration of FVIII:C (Cmax)
Time frame: Pre-dose and post dose up to 8 days.
Measured by aPTT Clotting Assay.
Time required for the concentration of the drug to reach half of its original value (T1/2)
Time frame: Pre-dose and post dose up to 8 days
Measured by aPTT Clotting Assay.
Area Under the Curve to Infinity (AUC)
Time frame: Pre-dose and post dose up to 8 days.
Measured by aPTT Clotting Assay.
The measure of the efficiency of the body to remove the drug and the unit is the volume of the plasma or blood cleared of drug per unit time (CL).
Time frame: Pre-dose and post dose up to 8 days.
Measured by aPTT Clotting Assay.
2 secondary outcomes
Number of participants with treatment-related adverse events as assessed by CTCAE V5.0.
Time frame: Post dose up to 32 days.
Development of Inhibitor
Time frame: Pre-dose and post dose up to 32 days.
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 22 Aug 2023.
- Individual participant dataSharing planned: No
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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.
Runhui Wu, PhD principal investigator
Beijing Children's Hospital
Xiaoling Wang, MA.Sc principal investigator
Beijing Children's Hospital
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ADVATE and FRSW107 in people with Hemophilia A, run by Hangzhou Gensciences Biopharmaceutical Co., Ltd. at 6 sites in China.
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 3 inclusion and 12 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 22 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in China. 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?
Maximum measured concentration of FVIII:C (Cmax) (time frame: Pre-dose and post dose up to 8 days.). The study lists 4 primary outcomes in all. 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 22 Aug 2023.