Hemophilia APhase 3NCT05707351
A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A
A Phase 3 study testing Adynovate in people with Hemophilia A, run by Takeda at 12 sites in China.
Trial summary
The main aim of the study is to determine how well Adynovate works to decrease bleeding in previously treated Chinese men and boys with severe hemophilia A when given prophylactically. Read more about this study
- Who is it for?
- Ages 12 to 65 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Adynovate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- 12 sitesChina
- Study dates
- 27 Mar 2023to 5 Sep 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Participant and/or legally authorized representative must voluntarily sign a written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the Participant. For the participants less than (<) 18 years old, participants will give assent AND their parents/legally authorized representative should sign the ICF accordingly.
- Participant and/or legally authorized representative understands and is willing and able to comply with all requirements of the study protocol.
- Participant should be ethnic Chinese.
- Participant is 12 to 65 years of age at screening and male.
- Participant has severe hemophilia A (FVIII clotting activity <1 percent [%]) as confirmed by the central laboratory at screening after a washout period of at least 72 to 96 hours.
- The last on-demand or prophylactic treatment received is within 3 months before screening.
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- Participant has documented previous treatment with plasma-derived FVIII concentrates or recombinant FVIII for greater than (>) 150 EDs.
- Participant is human immunodeficiency virus (HIV)-negative, or HIV-positive with stable disease and CD4+ count greater than or equal to (>=) 200 cells per cubic millimeter (/mm^3).
- Participant is hepatitis C virus (HCV) negative by antibody testing (if positive, additional polymerase chain reaction testing will be performed to confirm), as confirmed at screening; or HCV-positive with chronic stable hepatitis, as assessed by the investigator.
Who cannot join
- Participant has detectable FVIII inhibitory antibodies (>=0.6 Bethesda units [BU] per milliliter [/mL] using the Nijmegen modification of the Bethesda assay) as confirmed by the central laboratory at screening.
- Participant has a confirmed history of FVIII inhibitory antibodies (>=0.6 BU using the Nijmegen modification of the Bethesda assay or >=0.6 BU using the Bethesda assay) at any time prior to screening.
- Participant has a known hypersensitivity to Adynovate or ADVATE or any of the components of the study drugs, such as mouse or hamster proteins, or other FVIII products.
- Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (example, qualitative platelet defect or von Willebrand's disease).
- Participant has severe hepatic dysfunction (example, >=5 times the upper limit of normal [ULN] for alanine aminotransferase [ALT] or aspartate aminotransferase [AST], a recent or persistent international normalized ratio [INR] >1.5, as confirmed by the local laboratory at screening).
- Participant has severe renal impairment (serum creatinine >1.5 times the ULN) as confirmed by the local laboratory at screening.
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- Participant is planned or likely to undergo major surgery during the study period.
- Participant has current or recent (<30 days) use of other PEGylated drugs before study participation or scheduled use of such drugs during study participation.
- Participant has received emicizumab therapy within 6 months of screening.
- Participant is currently receiving, or scheduled to receive during the study, an immunomodulating drug (example, systemic corticosteroid agent at a dose equivalent to hydrocortisone >10 milligram per day [mg/day], or alpha-interferon) other than antiretroviral chemotherapy.
- Participant has participated in another clinical study involving the use of an investigational product (IP) other than Adynovate or an investigational device within 30 days before the screening visit or is scheduled to participate in another clinical study involving an IP or investigational device during this study.
- Participant has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
- Participant, in the opinion of the investigator, is unable or unwilling to comply with the study protocol.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
China12 sites
Beijing Children's Hospital, Capital Medical University
Beijing
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing
Xiangya Hospital of Central South University
Changsha
Fujian Medical University Union Hospital
Fuzhou
Nanfang Hospital Southern Medical University
Guangzhou
Anhui Province Hospital
Hefei
Jinan Central Hospital
Jinan
Shenzhen Second People's Hospital
Shenzhen
The First Affiliated Hospital of Soochow University
Suzhou
Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences
Tianjin
Tongji Hospital Tongji Medical College Huazhong University of Science and Technology
Wuhan
Union Hospital Tongji Medical College Huazhong University of Science and Technology
Wuhan
About this study
Official title: A Phase 3, Prospective, Multicenter, Open-label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (ADYNOVATE) Administered for Prophylaxis and Treatment of Bleeding in Chinese Previously Treated Patients With Severe Hemophilia A (FVIII <1%)
The main aim of the study is to determine how well Adynovate works to decrease bleeding in previously treated Chinese men and boys with severe hemophilia A when given prophylactically.
Participants will be treated with Adynovate twice a week for 26 weeks or until participants have received 50 days of treatment with Adynovate (whichever takes longer). Participants will need to visit their study clinic several times during their participation.
- Conditions
- Hemophilia A
- Being tested
- Adynovate
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AdynovateBiological
Also called Antihemophilic Factor (recombinant) PEGylated, Rurioctocog Alfa Pegol
Adynovate was injected intravenously using an appropriately sized syringe as a bolus infusion over a period of less than or equal to (<=) 5 minutes (maximum infusion rate, 10 milliliters per minute [mL/min]).
| Study group | Role | What they receive |
|---|---|---|
| Adynovate 45±5 IU/kg | Experimental | Participants received prophylactic treatment with Adynovate (45 [±5] IU/kg), infusion, IV, twice weekly, for at least 50 EDs, or approximately 28 weeks. Receives: Biological: Adynovate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Annualized Bleeding Rates (ABR)
Total ABR was defined as the number of treated and non-treated bleeding episodes (BEs) that occurred during the treatment period, calculated as, ABR= number of unique bleeds during treatment period/(length of treatment period [days]/365.25). Total ABR for all BEs, spontaneous or traumatic, recorded in the participant's electronic diary and/or recorded in the physician/nurse/study site notes were reported.
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Secondary outcomes (28)
ABR Based on Bleeding Site
ABR= number of unique bleeds during treatment period/(length of treatment period [days]/365.25). ABR for BEs based on bleeding site: joint or non-joint, recorded in the participant's electronic diary and/or recorded in the physician/nurse/study site notes were reported.
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
ABR Based on Bleeding Cause
ABR= number of unique bleeds during treatment period/(length of treatment period [days]/365.25). ABR for BEs based on bleeding cause: spontaneous/unknown or injury, recorded in the participant's electronic diary and/or recorded in the physician/nurse/study site notes were reported.
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Adynovate Infusions Per Week During the Prophylactic Treatment Period
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Adynovate Infusions Per Month During the Prophylactic Treatment Period
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Weight-adjusted Consumption of Adynovate Per Week During the Prophylactic Treatment Period
Weight-adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion.
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Weight-adjusted Consumption of Adynovate Per Month During the Prophylactic Treatment Period
Weight-adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion.
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Percentage of Participants With Zero Bleeding Episodes During the Study
Percentages were rounded off to the nearest single decimal place.
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Average Time Interval Between Bleeding Episodes (BEs)
Average time interval between bleeding episodes (days)= Length of treatment period (days)/ Number of unique bleeds during treatment period. Average time interval was computed for participants with more than 1 unique BEs.
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Bleeding Events in Each Category of Hemostatic Efficacy Rating at Resolution of Breakthrough Bleeding Episode
Hemostatic efficacy for treatment of BEs was rated on 4-point Likert scale as: excellent=full relief of pain and cessation of objective signs of bleeding after a single infusion, no additional infusion is required for the control of bleeding and administration of further infusion to maintain hemostasis would not affect the scoring; good=definite pain relief and/or improvement in signs of bleeding after a single infusion, possibly requires more than 2 infusions for complete resolution and administration of further infusion to maintain hemostasis would not affect the scoring; fair=probable and/or slight relief of pain and slight improvement in signs of bleeding after a single infusion, required multiple infusions for complete resolution; none=no improvement of signs or symptoms or conditions worsen. Missing indicates the number of unique bleeding episodes without any overall hemostatic efficacy rating at resolution of breakthrough bleeding episode.
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Adynovate Infusions Per Bleeding Episode
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Weight-adjusted Consumption of Adynovate Per Bleeding Episode
Weight-adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion.
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Minor Surgeries With Hemostatic Efficacy Based on Global Hemostatic Efficacy Assessment (GHEA) Score as Assessed by the Operating Surgeon/Investigator
GHEA score consisted of 3 individual rating scales: (1) Intra-operative Efficacy Assessment Scale, (2) Post-operative Efficacy Assessment Scale, and (3) Peri-operative Efficacy Assessment Scale. Each rating scale is based on 4 points scale ranging from: 3 (Excellent), 2 (Good), 1 (Fair), and 0 (None). The scores of 3 individual ratings scales were added together to form a GHEA score. Total score ranged from 0 to 9, where scores evaluate as: excellent (7 to 9), good (5 to 7), fair (3 to 4), and none (0 to 2). For a GHEA score of 7 to be rated "excellent" no individual assessment scores could be less than (<) 2 and at least 1 assessment score had to be equal to (=) 3; otherwise a score of 7 was rated "good".
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Volume of Actual and Predicted Intra-operative and Post-operative Blood Loss After the Surgery as Assessed by the Operating Surgeon/Investigator
Measured: Post-operative: Day 1 and at discharge Week 26
Number of Participants Who Required Perioperative Transfusion of Blood, Red Blood Cells, Platelets, and Other Blood Products
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Daily Intra-Operative and Post-Operative Weight-Adjusted Consumption Dose of Adynovate
Measured: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Treatment-emergent Adverse Events (Serious TEAEs)
An adverse event (AE): any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to medicinal product. TEAE: any AE either reported for the first time or worsening of a pre-existing event after first dose of study drug and within 30 days of the last administration of study drug. Serious TEAEs: any untoward medical occurrence that: 1) results in death, 2) is life-threatening, 3) requires inpatient hospitalization or prolongation of existing hospitalization, 4) results in persistent or significant disability/incapacity, 5) leads to a congenital anomaly/birth defect in the offspring of the participant or 6) is a medically important.
Measured: Up to approximately 28 weeks
Number of Participants With Confirmed Inhibitory Antibodies to Factor VIII (FVIII), Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies to Adynovate and Chinese Hamster Ovary (CHO) Protein
Measured: Up to approximately 28 weeks
FVIII Activity Level in Plasma Assessed by a 1-stage Clotting Assay
As per planned analysis, data for this outcome measure was collected and reported for initial pharmacokinetic (PK) assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit. FVIII activity level reported was corrected for pre-infusion measurement.
Measured: Day -1 and Week 20: pre-infusion, post-infusion at multiple time-points up to 96 hours
Incremental Recovery Over Time During Adynovate Prophylactic Treatment
Incremental recovery (IR) was calculated as IR (international units per deciliter)/(international units per kilogram [(IU/dL)/(IU/kg)] = [PostFVIII (IU/dL)-PreFVIII (IU/dL)]/Weight Adjusted Dose (IU/kg).
Measured: Baseline, Week 6, and Study Completion (approximately Week 28)
Pre-dose Level of FVIII Activity in Plasma
Measured: Baseline, Weeks 2, 6, 12, and Study Completion (approximately Week 28): Within 30 minutes pre-infusion
Pre-dose Level of FVIII Antigen in Plasma
IU/mL stands for international units per milliliter.
Measured: Baseline, Weeks 2, 6, 12, 20, and Study Completion (approximately Week 28): Within 30 minutes pre-infusion
Pre-dose Level of Von Willebrand Factor (VWF) Antigen in Plasma
Measured: Baseline, Weeks 2, 6, 12, 20, and Study Completion (approximately Week 28): Within 30 minutes pre-infusion
Clearance (CL) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
Clearance reported was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit. [(dL/h)/kg] stands for deciliters per hour per kilogram.
Measured: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Volume of Distribution for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
Volume of distribution was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit.
Measured: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Area Under the Concentration Versus Time Curve From 0 to 96 Hours (AUC0-96) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
AUC0-96 was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit. h*IU/dL stands for hour*international units per deciliter.
Measured: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Maximum Concentration (Cmax) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
Cmax was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit.
Measured: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Pre-dose Concentration (Cpredose) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
Cpredose was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit.
Measured: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Terminal Phase Elimination Half-life (T1/2) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
T1/2 was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit.
Measured: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2023
- Study start27 Mar 2023
- Primary completion5 Sep 2024
- Study completion5 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05707351
- Sponsor's protocol ID
- TAK-660-3001
- Other IDs
- 2023-000502-26, 2023
- Status last verified
- Apr 2025
- First submitted
- 23 Jan 2023
- Last updated
- 1 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Prospective, Multicenter, Open-label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (ADYNOVATE) Administered for Prophylaxis and Treatment of Bleeding in Chinese Previously Treated Patients With Severe Hemophilia A (FVIII <1%)
- ClinicalTrials.gov ID
- NCT05707351
- Acronym / sponsor ID
- TAK-660-3001
- Conditions
- Hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 27 Mar 2023 (actual)
- Primary completion
- 5 Sep 2024 (actual)
- Study completion
- 5 Sep 2024 (actual)
- First posted
- 31 Jan 2023 (actual)
- Results
- Posted 1 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main aim of the study is to determine how well Adynovate works to decrease bleeding in previously treated Chinese men and boys with severe hemophilia A when given prophylactically.
Participants will be treated with Adynovate twice a week for 26 weeks or until participants have received 50 days of treatment with Adynovate (whichever takes longer). Participants will need to visit their study clinic several times during their participation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adynovate 45±5 IU/kg | Experimental | Biological: AdynovateParticipants received prophylactic treatment with Adynovate (45 [±5] IU/kg), infusion, IV, twice weekly, for at least 50 EDs, or approximately 28 weeks. |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Participant and/or legally authorized representative must voluntarily sign a written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the Participant. For the participants less than (<) 18 years old, participants will give assent AND their parents/legally authorized representative should sign the ICF accordingly.
- Participant and/or legally authorized representative understands and is willing and able to comply with all requirements of the study protocol.
- Participant should be ethnic Chinese.
- Participant is 12 to 65 years of age at screening and male.
- Participant has severe hemophilia A (FVIII clotting activity <1 percent [%]) as confirmed by the central laboratory at screening after a washout period of at least 72 to 96 hours.
Key exclusion criteria (13 listed)
- Participant has detectable FVIII inhibitory antibodies (>=0.6 Bethesda units [BU] per milliliter [/mL] using the Nijmegen modification of the Bethesda assay) as confirmed by the central laboratory at screening.
- Participant has a confirmed history of FVIII inhibitory antibodies (>=0.6 BU using the Nijmegen modification of the Bethesda assay or >=0.6 BU using the Bethesda assay) at any time prior to screening.
- Participant has a known hypersensitivity to Adynovate or ADVATE or any of the components of the study drugs, such as mouse or hamster proteins, or other FVIII products.
- Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (example, qualitative platelet defect or von Willebrand's disease).
- Participant has severe hepatic dysfunction (example, >=5 times the upper limit of normal [ULN] for alanine aminotransferase [ALT] or aspartate aminotransferase [AST], a recent or persistent international normalized ratio [INR] >1.5, as confirmed by the local laboratory at screening).
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 |
|---|---|---|
| AdynovateAlso: Antihemophilic Factor (recombinant) PEGylated, Rurioctocog Alfa PegolAdynovate was injected intravenously using an appropriately sized syringe as a bolus infusion over a period of less than or equal to (<=) 5 minutes (maximum infusion rate, 10 milliliters per minute [mL/min]). | Biological | Adynovate 45±5 IU/kg |
Primary outcome
Total Annualized Bleeding Rates (ABR)
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Total ABR was defined as the number of treated and non-treated bleeding episodes (BEs) that occurred during the treatment period, calculated as, ABR= number of unique bleeds during treatment period/(length of treatment period [days]/365.25). Total ABR for all BEs, spontaneous or traumatic, recorded in the participant's electronic diary and/or recorded in the physician/nurse/study site notes were reported.
28 secondary outcomes
ABR Based on Bleeding Site
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
ABR Based on Bleeding Cause
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Adynovate Infusions Per Week During the Prophylactic Treatment Period
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Adynovate Infusions Per Month During the Prophylactic Treatment Period
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Weight-adjusted Consumption of Adynovate Per Week During the Prophylactic Treatment Period
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Weight-adjusted Consumption of Adynovate Per Month During the Prophylactic Treatment Period
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Percentage of Participants With Zero Bleeding Episodes During the Study
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Average Time Interval Between Bleeding Episodes (BEs)
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Bleeding Events in Each Category of Hemostatic Efficacy Rating at Resolution of Breakthrough Bleeding Episode
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Adynovate Infusions Per Bleeding Episode
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Weight-adjusted Consumption of Adynovate Per Bleeding Episode
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Minor Surgeries With Hemostatic Efficacy Based on Global Hemostatic Efficacy Assessment (GHEA) Score as Assessed by the Operating Surgeon/Investigator
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Volume of Actual and Predicted Intra-operative and Post-operative Blood Loss After the Surgery as Assessed by the Operating Surgeon/Investigator
Time frame: Post-operative: Day 1 and at discharge Week 26
Number of Participants Who Required Perioperative Transfusion of Blood, Red Blood Cells, Platelets, and Other Blood Products
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Daily Intra-Operative and Post-Operative Weight-Adjusted Consumption Dose of Adynovate
Time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Treatment-emergent Adverse Events (Serious TEAEs)
Time frame: Up to approximately 28 weeks
Number of Participants With Confirmed Inhibitory Antibodies to Factor VIII (FVIII), Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies to Adynovate and Chinese Hamster Ovary (CHO) Protein
Time frame: Up to approximately 28 weeks
FVIII Activity Level in Plasma Assessed by a 1-stage Clotting Assay
Time frame: Day -1 and Week 20: pre-infusion, post-infusion at multiple time-points up to 96 hours
Incremental Recovery Over Time During Adynovate Prophylactic Treatment
Time frame: Baseline, Week 6, and Study Completion (approximately Week 28)
Pre-dose Level of FVIII Activity in Plasma
Time frame: Baseline, Weeks 2, 6, 12, and Study Completion (approximately Week 28): Within 30 minutes pre-infusion
Pre-dose Level of FVIII Antigen in Plasma
Time frame: Baseline, Weeks 2, 6, 12, 20, and Study Completion (approximately Week 28): Within 30 minutes pre-infusion
Pre-dose Level of Von Willebrand Factor (VWF) Antigen in Plasma
Time frame: Baseline, Weeks 2, 6, 12, 20, and Study Completion (approximately Week 28): Within 30 minutes pre-infusion
Clearance (CL) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
Time frame: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Volume of Distribution for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
Time frame: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Area Under the Concentration Versus Time Curve From 0 to 96 Hours (AUC0-96) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
Time frame: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Maximum Concentration (Cmax) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
Time frame: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Pre-dose Concentration (Cpredose) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
Time frame: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Terminal Phase Elimination Half-life (T1/2) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
Time frame: Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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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.
Medical Director study director
Takeda
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Adynovate in people with Hemophilia A, run by Takeda at 12 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 9 inclusion and 13 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 1 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Total Annualized Bleeding Rates (ABR) (time frame: Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)). 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 1 May 2025.