A Single-Arm, Open-Label, Exploratory Clinical Study to Evaluate the Safety and Efficacy of Elranatamab for Inhibitor Eradication in Patients With Moderate-to-Severe Hemophilia A With Inhibitors and Poor Prognosis for Inhibitor Eradication
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 20
- 主要终点
- Incidence of Treatment-Emergent Adverse Events (AES)
研究概览
简要总结
This is a prospective, single-arm, open-label, exploratory clinical study designed to evaluate the safety and efficacy of Elranatamab for inhibitor eradication in adults with moderate-to-severe hemophilia A with inhibitors. A total of 15-20 patients aged ≥18 years will be enrolled. Elranatamab will be administered with a stepwise dose-escalation regimen of 12 mg on Day 1 and 32 mg on Day 4 during Week 1, followed by the target dose of 38 mg once weekly during Weeks 2-3. Patients will then enter a 4-week follow-up period. If the inhibitor titer decreases by <20% from baseline at Week 3, two additional weekly doses of 38 mg may be administered before follow-up. Treatment response will be assessed according to changes in inhibitor titers following treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients with moderate-to-severe hemophilia A (factor VIII activity <2%).
- •Aged 18 to 65 years, inclusive.
- •Positive factor VIII inhibitor detected on at least two consecutive occasions (inhibitor titer >0.6 BU/mL).
- •Baseline factor VIII inhibitor titer >10 BU/mL at the time of enrollment.
排除标准
- •Known hypersensitivity to Elranatamab or any of its excipients.
- •Presence of other autoimmune diseases or a need for immunosuppressive therapy for reasons unrelated to this study.
- •History of malignancy within 5 years prior to screening, with the exception of adequately treated carcinoma in situ of the cervix or non-metastatic cutaneous squamous cell carcinoma or basal cell carcinoma.
- •History of severe recurrent or chronic infections, or acute infection requiring systemic treatment with antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungal agents within 4 weeks before the first dose or during the screening period, or superficial skin infection requiring systemic therapy within 1 week before the first dose.
- •Clinically significant laboratory abnormalities at screening, including:
- •Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3 × the upper limit of normal (ULN).
- •Total bilirubin >1.5 × ULN (subjects with documented Gilbert syndrome are not excluded based on this criterion);
- •Absolute neutrophil count <1,500/mm³;
- •Hemoglobin <9 g/dL or immunoglobulin G (IgG) <500 mg/dL;
- •Absolute lymphocyte count <500/mm³;
- •Creatinine clearance (CrCl) <30 mL/min.
- •Positive test for HIV antibody or syphilis antibody.
- •Positive hepatitis B surface antigen (HBsAg); or positive hepatitis B core antibody (anti-HBc) with detectable HBV DNA by polymerase chain reaction (PCR). Subjects positive for hepatitis C virus (HCV) antibody are excluded.
- •Women who are pregnant or breastfeeding, or who plan to become pregnant or breastfeed during the study; men whose partners plan to become pregnant during the study.
- •Subjects with psychiatric disorders that impair their ability to provide informed consent or comply with study procedures and follow-up.
- •Subjects with unresolved toxicities from prior therapies before study participation.
- •Any other condition that, in the opinion of the investigator, would make the subject unsuitable for participation in the study.
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events (AES)
时间窗: 1 year
AES was assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0.
次要结局
- The proportion of patients who achieve successful inhibitor eradication(1 year)