An Open-label, Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 in Subjects With Thrombotic Microangiopathy Secondary to Systemic Lupus Erythematosus
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 24
- 主要终点
- Parts 1 and 2: Number of participants with Treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs) and Adverse events of special interest (AESIs)
研究概览
简要总结
This study will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KP104 in participants with systemic lupus erythematosus (SLE)-Thrombotic microangiopathy (TMA). The study consists of 2 parts: Part 1 (Dose Optimization) and Part 2 (Proof of Concept). All participants will receive KP104 in combination with standard of care (SOC) for SLE-TMA.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Meets criteria for SLE per the 2019 European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) criteria.
- •Decrease in platelet count to less than (<)150,000/microliters (mcL).
- •Abnormal renal function.
- •Females of childbearing potential with negative pregnancy test and males must agree to practice effective contraception from Screening until 28 days after the End of study (EOS) visit.
- •Willing and able to provide informed consent.
- •Evidence of microangiopathic hemolytic anemia
排除标准
- •Diagnosis of other TMA syndromes.
- •A renal biopsy within 7 days of screening that shows exclusively chronic changes of TMA.
- •Positive Coombs test at the time of TMA diagnosis.
- •Active or unresolved Neisseria meningitidis infection at screening.
- •Only key inclusion and exclusion criteria have been included.
研究组 & 干预措施
Part 1: Dose Optimization Cohort 1, Dose 1
Participants will be administered with KP104 as a weekly maintenance dose for 24 Weeks. After the last participant completes 6 weeks of treatment, all available data, including safety, PK, PD, and modeling results, will be reviewed by the Internal Data Review Committee (IDRC) to determine Dosing Regimen 2
干预措施: KP104 (Drug)
Part 1: Dose Optimization Cohort 2, Dose 2
Participants will be administered with KP104 dose regimen 2 for 24 Weeks. After the last participant completes 6 weeks of treatment, all available data, including safety, PK, PD, and modeling results, will be reviewed by the IDRC to determine Dosing Regimen 3.
干预措施: KP104 (Drug)
Part 1: Dose Optimization Cohort 3, Dose 3
Participants will be administered with KP104 dose regimen 3 for 24 Weeks. After the last participant completes 6 weeks of treatment, all available data, including safety, PK, PD, and modeling results, will be reviewed by IDRC to determine the Optimal biologic dose (OBD) for Part 2.
干预措施: KP104 (Drug)
Part 2: OBD Cohort, Dose 4
Participants will be administered with KP104 OBD for 24 Weeks.
干预措施: KP104 (Drug)
结局指标
主要结局
Parts 1 and 2: Number of participants with Treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs) and Adverse events of special interest (AESIs)
时间窗: Up to 24 weeks
Part 2: Percent change from Baseline in platelet count
时间窗: Baseline (Day 1) and up to Week 12
Part 2: Percent change from Baseline in serum lactate dehydrogenase (LDH) levels
时间窗: Baseline (Day 1) and up to Week 12
次要结局
未报告次要终点