Hemophilia ANCT03747653
A Study to Evaluate the Pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
An observational study of Hemophilia A, run by Kaifeng Pharmaceutical (Group) Co., Ltd. at 5 sites in China.
Trial summary
Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection at two dose levels in patients with hemophilia A. Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in patients with hemophilia A. Read more about this study
- Who is it for?
- Ages 12 to 60 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- ADVATE and FRSW107
- Type of treatment
- Injection or infusion
- How many people
- 12 participants
- Where
- 5 sitesChina
- Study dates
- 8 Mar 2019to 31 May 2020
- Phase
- Observational
- 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 60 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Adolescent and Adult patients with Hemophilia A from clinical registration.
Who may be able to join
- 12 years to 60 years, male.
- The activity of the coagulation factor VIII (FVIII:C) < 2%, and previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry.
- Non-immune deficiency (CD4 > 200/μL).
- Non-acute hemorrhagic state.
- No history of a positive inhibitor test (< 0.6 BU) or clinical signs of decreased response to FVIII administrations. No Family history of inhibitors.
- Platelet count > 100,000 platelets/μL.
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- Normal prothrombin time or INR < 1.3.
- Normal thrombin time (TT).
- Normal previous results of vWF antigen examination.
- Negative lupus anticoagulant .
- Capable of understanding and willing to comply with the conditions of the protocol have read (patient and/or guardian).
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 IgG2 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 4 days prior screening or within 72 h prior to administration.
- Patients with severe heart disease, including myocardial infarction, heart failure (III or higher level).
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- Clinically significant of other systematic diseases: alcoholism, drug abuse, mental disorders and mental retardation.
- Significant hepatic or renal impairment (ALT and AST > 2×ULN; serum bilirubin level > 3 × upper limit of normal (ULN) , BUN > 2×ULN, Cr > 2.0 mg/dL).
- 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 1 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.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
China5 sites
Hematology Department, Beijing Children's Hospital, Capital Medical University
Beijing, 100045
Fujian Medical University Union Hospital
Fuzhou, Fujian, 350000
Southern Medical University Nanfang Hospital
Guangzhou, Guangzhou, 510515
Jinan Central Hospital
Jinan, Shandong, 250013
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, 300020
About this study
Official title: A Phase I, Multicentre, Open-label, Self-control Study to Evaluate the Pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A
Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection at two dose levels in patients with hemophilia A.
Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in patients with hemophilia A.
- Conditions
- Hemophilia A
- Being tested
- ADVATE, FRSW107
- Keywords
- Hemophilia A, Pharmacokinetics, Factor VIII-Fc Fusion Protein, Adolescent and Adult Patients, Phase I, 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
Patients will be administered a single dose of ADVATE.
FRSW107Drug
Patients will be administered a single dose of FRSW107 for Injection.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Participants will receive a single intravenous (i.v.) injection of ADVATE followed by a single intravenous (i.v.) injection of Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection (FRSW107) at a low dose. Receives: Drug: ADVATE; Drug: FRSW107 | |
| Arm 2 | Participants will receive a single intravenous (i.v.) injection of ADVATE followed by a single intravenous (i.v.) injection of Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection (FRSW107) at a high dose. 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 the One-stage aPTT Clotting Assay.
Measured: Pre-dose and post dose of FRSW107 up to 10 days.
Time required for the concentration of the drug to reach half of its original value (T1/2).
Measured by the One-stage aPTT Clotting Assay.
Measured: Pre-dose and post dose of FRSW107 up to 10 days.
Area Under the Curve to Infinity (AUC).
Measured by the One-stage aPTT Clotting Assay.
Measured: Pre-dose and post dose of FRSW107 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 the One-stage aPTT Clotting Assay.
Measured: Pre-dose and post dose of FRSW107 up to 10 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 of FRSW107 up to 28.
Development of Inhibitor.
Measured by the Nijmegen-Modified Bethesda Assay.
Measured: Pre-dose and post dose of FRSW107 up to 28 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2018
- Study start8 Mar 2019
- Primary completion31 May 2020 (estimated)
- Study completion31 May 2020 (estimated)
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
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kaifeng Pharmaceutical (Group) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Beijing Furen Biomedical Research Institute Co., Ltd.
- 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
- NCT03747653
- Sponsor's protocol ID
- CTR20182090
- Status last verified
- May 2020
- First submitted
- 12 Nov 2018
- Last updated
- 8 May 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Multicentre, Open-label, Self-control Study to Evaluate the Pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A
- ClinicalTrials.gov ID
- NCT03747653
- Acronym / sponsor ID
- CTR20182090
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, Pharmacokinetics, Factor VIII-Fc Fusion Protein, Adolescent and Adult Patients, Phase I, Safety and Tolerability
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Kaifeng Pharmaceutical (Group) Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Beijing Furen Biomedical Research Institute Co., Ltd.
- Study start
- 8 Mar 2019 (actual)
- Primary completion
- 31 May 2020 (estimated)
- Study completion
- 31 May 2020 (estimated)
- First posted
- 20 Nov 2018 (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 at two dose levels in patients with hemophilia A.
Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in patients with hemophilia A.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | – | Drug: ADVATE, Drug: FRSW107Participants will receive a single intravenous (i.v.) injection of ADVATE followed by a single intravenous (i.v.) injection of Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection (FRSW107) at a low dose. |
| Arm 2 | – | Drug: ADVATE, Drug: FRSW107Participants will receive a single intravenous (i.v.) injection of ADVATE followed by a single intravenous (i.v.) injection of Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection (FRSW107) at a high dose. |
Who may qualify
- Ages
- 12 to 60 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Adolescent and Adult patients with Hemophilia A from clinical registration.
Key inclusion criteria (11 listed)
- 12 years to 60 years, male.
- The activity of the coagulation factor VIII (FVIII:C) < 2%, and previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry.
- Non-immune deficiency (CD4 > 200/μL).
- Non-acute hemorrhagic state.
- No history of a positive inhibitor test (< 0.6 BU) or clinical signs of decreased response to FVIII administrations. No Family history of inhibitors.
Key exclusion criteria (14 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 IgG2 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 4 days prior screening or 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 |
|---|---|---|
| ADVATEPatients will be administered a single dose of ADVATE. | Drug | Arm 1, Arm 2 |
| FRSW107Patients will be administered a single dose of FRSW107 for Injection. | Drug | Arm 1, Arm 2 |
Primary outcome
Maximum measured concentration of FVIII:C (Cmax).
Time frame: Pre-dose and post dose of FRSW107 up to 10 days.
Measured by the One-stage 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 of FRSW107 up to 10 days.
Measured by the One-stage aPTT Clotting Assay.
Area Under the Curve to Infinity (AUC).
Time frame: Pre-dose and post dose of FRSW107 up to 10 days.
Measured by the One-stage 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 of FRSW107 up to 10 days.
Measured by the One-stage aPTT Clotting Assay.
2 secondary outcomes
Number of participants with treatment-related adverse events as assessed by CTCAE V5.0.
Time frame: Post dose of FRSW107 up to 28.
Development of Inhibitor.
Time frame: Pre-dose and post dose of FRSW107 up to 28 days.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2020.
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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?
An observational study of Hemophilia A, run by Kaifeng Pharmaceutical (Group) 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 11 inclusion and 14 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: status not confirmed, as of the sponsor's last update on 8 May 2020. 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.
Are results available for this study?
Results are not available in the source record.
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 of FRSW107 up to 10 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 8 May 2020.