Clinical Trials List
Protocol NumberLPS18168
Active
2025-05-30 - 2027-07-12
Phase III
Recruiting3
ICD-10D66
Hereditary factor VIII deficiency
ICD-9286.0
Congenital factor VIII disorder
SYNOVIIIUS: Prospective Interventional Study of Effectiveness of Efanesoctocog Alfa Prophylaxis on Synovial Hypertrophy in Patients with Hemophilia A
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Sponsor
Sanofi Taiwan Co., Ltd.
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Trial scale
Multi-Regional Multi-Center
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Update
2026/02/01
Investigators and Locations
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Recruiting
Principal Investigator
Shyh-Shin Chiou
Co-Principal Investigator
- 許琬宜 無
- 廖優美 無
- Pei-Chin Lin 無
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Hemophilia A
Objectives
The purpose of this study is to investigate the effect of efanesoctocog alfa as a prophylactic treatment on synovial hypertrophy in patients with hemophilia A. Prophylactic treatment refers to a medication or therapeutic approach used to prevent the occurrence of a disease or condition. This study will evaluate the tolerability and potential adverse effects of the drug in order to assess the safety of efanesoctocog alfa.
Test Drug
Lyophilized powder for injection
Lyophilized powder for injection
Lyophilized powder for injection
Lyophilized powder for injection
Lyophilized powder for injection
Lyophilized powder for injection
Lyophilized powder for injection
Lyophilized powder for injection
Lyophilized powder for injection
Active Ingredient
Efanesoctocog alfa (BIVV001)
Dosage Form
243
243
243
243
243
243
243
243
243
Dosage
250 IU per vial
500 IU per vial
2000 IU per vial
3000 IU per vial
4000 IU per vial
500 IU per vial
2000 IU per vial
3000 IU per vial
4000 IU per vial
Endpoints
At Week 52, the proportion of joints showing improvement in synovitis domain scores on Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) assessment (i.e., a decrease of at least 1 point from baseline).
Inclution Criteria
Main Inclusion Criteria
Each patient must meet all of the following criteria to be eligible for inclusion in this study:
Diagnosed with moderate to severe hemophilia A at the time of consent/informed consent, defined as endogenous FVIII coagulation activity ≤ 5%.
Aged ≥ 12 years at the time of consent/informed consent.
Presence of pre-existing synovial hypertrophy at the time of consent/informed consent, defined as having at least one eligible joint* with a HEAD-US synovitis score of 1 or 2.
Has at least one eligible joint* that is not scheduled for major orthopedic intervention (e.g., arthroscopic synovectomy, radionuclide or chemical synovial therapy) during the study period, and has not undergone major orthopedic intervention within 3 months prior to the screening visit (Visit 1).
*An eligible joint is defined as a joint with pre-existing synovial hypertrophy, indicated by a HEAD-US synovitis score of 1 or 2, where synovial thickening is considered evidence of synovitis.
Has received a prescribed prophylactic hemophilia treatment regimen within 12 months prior to the baseline visit (Visit 2).
Able to understand the written Informed Consent Form (ICF)/Assent Form and provide a signed and witnessed written ICF/Assent, agreeing to comply with the study protocol requirements.
If male, no contraception measures are required to participate in this study.
If female, must be non-pregnant and non-lactating, and meet one of the following criteria:
Is a woman of non-childbearing potential (WONCBP), or
Is a woman of childbearing potential (WOCBP) who agrees to use an effective contraceptive method with a failure rate < 1% during the study treatment period (at least until Week 52 / End-of-Treatment visit), as described in the section “Contraception and Barrier Guidance and Pregnancy Data Collection.”
At the screening visit (Visit 1), WOCBP must have a negative serum pregnancy test result.
Each patient must meet all of the following criteria to be eligible for inclusion in this study:
Diagnosed with moderate to severe hemophilia A at the time of consent/informed consent, defined as endogenous FVIII coagulation activity ≤ 5%.
Aged ≥ 12 years at the time of consent/informed consent.
Presence of pre-existing synovial hypertrophy at the time of consent/informed consent, defined as having at least one eligible joint* with a HEAD-US synovitis score of 1 or 2.
Has at least one eligible joint* that is not scheduled for major orthopedic intervention (e.g., arthroscopic synovectomy, radionuclide or chemical synovial therapy) during the study period, and has not undergone major orthopedic intervention within 3 months prior to the screening visit (Visit 1).
*An eligible joint is defined as a joint with pre-existing synovial hypertrophy, indicated by a HEAD-US synovitis score of 1 or 2, where synovial thickening is considered evidence of synovitis.
Has received a prescribed prophylactic hemophilia treatment regimen within 12 months prior to the baseline visit (Visit 2).
Able to understand the written Informed Consent Form (ICF)/Assent Form and provide a signed and witnessed written ICF/Assent, agreeing to comply with the study protocol requirements.
If male, no contraception measures are required to participate in this study.
If female, must be non-pregnant and non-lactating, and meet one of the following criteria:
Is a woman of non-childbearing potential (WONCBP), or
Is a woman of childbearing potential (WOCBP) who agrees to use an effective contraceptive method with a failure rate < 1% during the study treatment period (at least until Week 52 / End-of-Treatment visit), as described in the section “Contraception and Barrier Guidance and Pregnancy Data Collection.”
At the screening visit (Visit 1), WOCBP must have a negative serum pregnancy test result.
Exclusion Criteria
Main Exclusion Criteria
Patients meeting any of the following criteria must not be enrolled in this study:
Has a diagnosed bleeding disorder other than hemophilia A at the time of consent/informed consent.
Currently receiving efanesoctocog alfa treatment.
Presence of FVIII inhibitors, defined as an inhibitor titer ≥ 0.60 BU/mL at the time of diagnosis.
Has received immune tolerance induction (ITI) therapy, as defined by Nakar and Shapiro (2019), within 2 years prior to the baseline visit (Visit 2).
Has participated in another interventional clinical trial or has been exposed to an investigational medicinal product within 3 months prior to screening.
Is currently institutionalized by regulatory or legal order (e.g., prisoners or patients under mandatory detention).
In the investigator’s judgment, is unsuitable for study participation for any reason, including medical or clinical conditions or potential non-compliance with study procedures.
Is an employee or family member of the investigator or study site personnel.
Is involved in any situation during or related to the conduct of the study that could raise ethical concerns.
Has a known hypersensitivity or allergic reaction to efanesoctocog alfa, its components, or any of its excipients, which in the opinion of the investigator precludes participation in this study.
Patients meeting any of the following criteria must not be enrolled in this study:
Has a diagnosed bleeding disorder other than hemophilia A at the time of consent/informed consent.
Currently receiving efanesoctocog alfa treatment.
Presence of FVIII inhibitors, defined as an inhibitor titer ≥ 0.60 BU/mL at the time of diagnosis.
Has received immune tolerance induction (ITI) therapy, as defined by Nakar and Shapiro (2019), within 2 years prior to the baseline visit (Visit 2).
Has participated in another interventional clinical trial or has been exposed to an investigational medicinal product within 3 months prior to screening.
Is currently institutionalized by regulatory or legal order (e.g., prisoners or patients under mandatory detention).
In the investigator’s judgment, is unsuitable for study participation for any reason, including medical or clinical conditions or potential non-compliance with study procedures.
Is an employee or family member of the investigator or study site personnel.
Is involved in any situation during or related to the conduct of the study that could raise ethical concerns.
Has a known hypersensitivity or allergic reaction to efanesoctocog alfa, its components, or any of its excipients, which in the opinion of the investigator precludes participation in this study.
The Estimated Number of Participants
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Taiwan
3 participants
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Global
35 participants
