NCT06941870招募中4 期
Prospective Interventional Study of Effectiveness of Efanesoctocog Alfa Prophylaxis on Synovial Hypertrophy in Patients With Hemophilia A
干预措施
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- Sanofi
- 入组人数
- 35
- 试验地点
- 13
- 主要终点
- Proportion of joints with improvement in the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovitis domain score
研究概览
简要总结
The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants with hemophilia A.
The study duration for each participant is approximately 12 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant is diagnosed with moderate to severe hemophilia A (defined as less than or equal to (≤)5 percent (%) of endogenous FVIII clotting activity) at the time of consent/assent.
- •Participant is more than or equal to (≥)12 years old at the time of consent/assent.
- •Participant has existing synovial hypertrophy, defined as at least 1 eligible* joint by the HEAD-US score (Synovitis score: 1 or 2) at the time of consent/assent.
- •Participant has at least 1 eligible* joint without planned future major orthopedic procedures (example, arthroscopic synovectomy, radioisotopic or chemical synoviorthesis), or major orthopedic procedures in the past 3 months prior to the screening visit (Visit 1).
- •*An eligible joint is a joint with existing synovial hypertrophy, as defined by a HEAD-US synovitis score of 1 or 2, considering hypertrophic synovium as an indication of the presence of synovitis Participant has received prophylactic treatment with hemophilia prophylaxis prescriptions in the last 12 months prior to the baseline visit (Visit 2).
- •Participant is capable of understanding the written Informed Consent Form (ICF)/assent form, provides a signed and witnessed written ICF/assent form, and agrees to comply with the protocol requirements.
- •If male, no contraceptive measures are required for this study.
- •If female, is not pregnant or breastfeeding, and one of the following conditions applies:
- •Is a women of nonchildbearing potential (WONCBP) Is a women of childbearing potential (WOCBP) and agrees to use a highly effective contraceptive method, with a failure rate of less than (<)1 percent during the study treatment period (at least until the Week 52/ End of Treatment [EoT] visit).
- •A WOCBP must have a negative serum pregnancy test at the screening visit (Visit 1)
排除标准
- •Participants are excluded from the study if any of the following criteria apply:
- •Has other associated clotting disorders at the time of consent/assent. Is already under efanesoctocog alfa treatment. Has a current diagnosis of an factor VIII (FVIII) inhibitor, defined as inhibitor titer ≥0.60 BU/mL.
- •Has ITI within the last 2 years prior to the baseline visit (Visit 2). Has been enrolled in a concurrent clinical interventional study or exposed to other investigational drug(s) within 3 months prior to screening for this study.
- •Is currently in an institution because of regulatory or legal order (that is, is a prisoner or a patient who is legally institutionalized).
- •Is not suitable for participation, whatever the reason, as judged by the investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.
- •Is an employee or family member of the investigator or site personnel. Is involved in a specific situation during study implementation or the course of the study that may raise ethics considerations.
- •Has hypersensitivity to efanesoctocog alfa or its components or any of its excipients that, in the opinion of the investigator, contraindicates participation in the study.
研究组 & 干预措施
Efanesoctocog Alfa
Experimental
Participants will receive Efanesoctocog Alfa at 50 international unit per kilogram (IU/kg) intravenous (IV) QW
干预措施: Efanesoctocog alfa (Drug)
结局指标
主要结局
Proportion of joints with improvement in the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovitis domain score
时间窗: From Baseline up to Week 52
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
次要结局
- The occurrence of Adverse Events (AEs), all AEs leading to treatment discontinuation, Serious Adverse Events (SAEs), and Adverse Event of Special Interest (AESIs)(Up to Week 52)
- Change from baseline in the distribution of joint HEAD-US synovitis score(From Baseline up to Week 52)
- Number of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened(From Baseline up to Week 52)
- Percentage of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened(From Baseline up to Week 52)
- Number of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened(From Baseline up to Week 52)
- Percentage of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened(From Baseline up to Week 52)
- Number of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened(From Baseline up to Week 52)
- Percentage of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened(From Baseline up to Week 52)
- Change from baseline in average HEAD-US synovitis domain score across all index joints(From Baseline up to Week 52)
- Change from baseline in total/ domain scores of the HEAD-US(From Baseline up to Week 26)
- Change from baseline in total/domain scores of the HJHS(From Baseline up to Week 52)
- Change from baseline in total/ domain scores of magnetic resonance imaging (MRI)(From Baseline up to Week 52)
- Change from baseline in patient-reported outcome (PRO)(From Baseline up to Week 52)
- Patient reported treatment preference and satisfaction(At Week 52)
- Change from baseline in annualized bleeding rate (ABR) (spontaneous, traumatic)(At Week 52)
- Change from baseline in annualized joint bleeding rate (AjBR) (spontaneous, traumatic)(At Week 52)
- Change from baseline in target joint resolution, recurrence, or development(At Week 52)
研究者
研究点 (13)
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