Severe Hemophilia APhase 2NCT05265286
A Study of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
A Phase 2 study testing FRSW117 in people with Severe Hemophilia A, run by Hangzhou Gensciences Biopharmaceutical Co., Ltd. at 5 sites in China.
Trial summary
Primary objective: To assess the pharmacokinetics, Safety and immunogenicity of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A(FRSW117) Secondary objectives: To assess Preliminary efficacy of Repeat Dosing of PEG Recombinant Human Coagulation… Read more about this study
- Who is it for?
- Ages 12 to 65 years, men only
- Conditions the study is for
- Severe Hemophilia A
- What is being tested?
- FRSW117
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 5 sitesChina
- Study dates
- 14 Apr 2022to 21 Aug 2022
- Phase
- Phase 2
- 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 12 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- The activity of the coagulation factor VIII (FVIII:C) < 1%.
- 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.• Significant hepatic or renal impairment (ALT and AST > 2×ULN; serum bilirubin level > 2 × upper limit of normal (ULN), Urea /BUN > 2×ULN, Cr > 176.8 µmol/L)
- 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
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- 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 screening
- 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
- Other protocol-defined inclusion/exclusion Criteria May Apply.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
China5 sites
The Affiliated Hospital of Qingdao University
Qingdao, Shandong, 266000
People'S Hospital of Rizhao
Rizhao, Shandong, 276800
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
Tianjin, Tianjin Municipality, 300020
Affiliated Hospital of Jiangnan University
Wuxi, Jiangsu, 214100
People'S Hospital of Zhengzhou
Zhengzhou, Henan, 450000
About this study
Official title: A Phase II, Multicentre, Open-label Study to Evaluate the Pharmacokinetic, Safety and Preliminary Efficacy of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Subjects With Severe Hemophilia A
Primary objective: To assess the pharmacokinetics, Safety and immunogenicity of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A(FRSW117) Secondary objectives: To assess Preliminary efficacy of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A.
- Conditions
- Severe Hemophilia A
- Being tested
- FRSW117
- Keywords
- Pharmacokinetics;, Safety;, immunogenicity;, Preliminary efficacy
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FRSW117Drug
Also called PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
Subjects of high dose group are being received four doses(50 IU/kg,once a week or every 7 days) of FRSW117.
Subjects of low dose group (40 IU/kg,once a week or every 7 days)are being received four doses of FRSW117.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 prophylaxis treatment | Experimental | Subjects of high dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively.
Subjects of low dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively.
All subjects are being received PK assessment in ED1 and ED4. Receives: Drug: FRSW117 |
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 One-Stage Clotting Assay
Measured: Pre-dose and post dose up to 10 days
Time required for the concentration of the drug to reach half of its original value (T1/2)
Measured by One-Stage Clotting Assay
Measured: Pre-dose and post dose up to 10 days
Area Under the Curve to Infinity (AUC)
Measured by One-Stage Clotting Assay
Measured: Pre-dose and post dose up to 10 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 One-Stage Clotting Assay
Measured: Pre-dose and post dose up to 10 days
Evaluation of the level of anti-PEG-rFⅧFc antibody production in participants
Measured: Pre-dose and post dose up to 36 days
Evaluation of the level of anti-PEG antibody production in participants
Measured: Pre-dose and post dose up to 36 days
Secondary outcomes (3)
Number of participants with treatment-related adverse events as assessed by CTCAE V5.0
Adverse events related to PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein according to Common Terminology Criteria for Adverse Events (CTCAE) NCI.V5.0.
Measured: Pre-dose and post dose up to 36 days
Development of Inhibitor
Measured by the Nijmegen-Modified Bethesda Assay
Measured: Pre-dose and post dose up to 36 days
score of bleeding symptoms and Vital signs
Response to treatment with PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for bleeding episodes, using the 4-point bleeding response scale
Measured: Pre-dose and post dose up to 36 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2022
- Study start14 Apr 2022
- Primary completion21 Aug 2022
- Study completion21 Aug 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Renchi Yang, PhD, Principal investigator, Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05265286
- Sponsor's protocol ID
- CTR20220283
- Status last verified
- May 2023
- First submitted
- 22 Feb 2022
- Last updated
- 15 May 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 15 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Multicentre, Open-label Study to Evaluate the Pharmacokinetic, Safety and Preliminary Efficacy of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Subjects With Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT05265286
- Acronym / sponsor ID
- CTR20220283
- Conditions
- Severe Hemophilia A
- Keywords
- Pharmacokinetics;, Safety;, immunogenicity;, Preliminary efficacy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 14 Apr 2022 (actual)
- Primary completion
- 21 Aug 2022 (actual)
- Study completion
- 21 Aug 2022 (actual)
- First posted
- 3 Mar 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, Safety and immunogenicity of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A(FRSW117) Secondary objectives: To assess Preliminary efficacy of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With 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 prophylaxis treatment | Experimental | Drug: FRSW117Subjects of high dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively. Subjects of low dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively. All subjects are being received PK assessment in ED1 and ED4. |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- The activity of the coagulation factor VIII (FVIII:C) < 1%.
- 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 (11 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.• Significant hepatic or renal impairment (ALT and AST > 2×ULN; serum bilirubin level > 2 × upper limit of normal (ULN), Urea /BUN > 2×ULN, Cr > 176.8 µmol/L)
- One or more clinically significant tests for Human Immunodeficiency Virus (HIV), Antisyphilitic spirulina (TPHA) and Hepatitis C Virus (HCV) Antibody
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 |
|---|---|---|
| FRSW117Also: PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for InjectionSubjects of high dose group are being received four doses(50 IU/kg,once a week or every 7 days) of FRSW117. Subjects of low dose group (40 IU/kg,once a week or every 7 days)are being received four doses of FRSW117. | Drug | Arm 1 prophylaxis treatment |
Primary outcome
Maximum measured concentration of FVIII:C (Cmax)
Time frame: Pre-dose and post dose up to 10 days
Measured by One-Stage 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 10 days
Measured by One-Stage Clotting Assay
Area Under the Curve to Infinity (AUC)
Time frame: Pre-dose and post dose up to 10 days
Measured by One-Stage 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 10 days
Measured by One-Stage Clotting Assay
Evaluation of the level of anti-PEG-rFⅧFc antibody production in participants
Time frame: Pre-dose and post dose up to 36 days
Evaluation of the level of anti-PEG antibody production in participants
Time frame: Pre-dose and post dose up to 36 days
3 secondary outcomes
Number of participants with treatment-related adverse events as assessed by CTCAE V5.0
Time frame: Pre-dose and post dose up to 36 days
Development of Inhibitor
Time frame: Pre-dose and post dose up to 36 days
score of bleeding symptoms and Vital signs
Time frame: Pre-dose and post dose up to 36 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 15 May 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.
Renchi Yang, PhD principal investigator
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing FRSW117 in people with Severe Hemophilia A, run by Hangzhou Gensciences Biopharmaceutical Co., Ltd. at 5 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 4 inclusion and 11 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 15 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 10 days). The study lists 6 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 15 May 2023.