A Phase 1B/2, Multicenter, Open-Label, Safety, Tolerability, and Activity Study of SYNT001 in Patients With Warm Autoimmune Hemolytic Anemia (WAIHA)
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
This main study objective was to evaluate the safety and tolerability of intravenous (IV) SYNT001 (ALXN1830) in participants with WAIHA.
详细描述
This study planned to evaluate 2 cohorts: Cohort 1, up to 8 participants to receive IV doses of ALXN1830 (SYNT001 Dose 1); Cohort 2, up to 12 participants to receive IV doses of ALXN1830 (SYNT001 Dose 2).
This study was terminated after the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy were characterized in participants with WAIHA in Cohort 1 (SYNT001 Dose 1), before any participants were enrolled in Cohort 2.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants had to meet the following criteria to be included:
- •Willing and able to read, understand, and sign an informed consent form
- •Confirmed diagnosis of WAIHA by enrolling physician
- •Must have used medically acceptable contraception
排除标准
- •Participants who met any of the following criteria were excluded:
- •Participant unable or unwilling to comply with the protocol
- •Active non-hematologic malignancy or history of non-hematologic malignancy in the 3 years prior to screening (exclusive of non-melanoma skin cancer and cervical cancer in situ)
- •Positive for human immunodeficiency virus or hepatitis C antibody
- •Positive for hepatitis B surface antigen
- •Any exposure to an investigational drug or device within the 30 days prior to screening
- •Intravenous immunoglobulin treatment within 30 days of screening
- •Plasmapheresis or immunoadsorption within 30 days of screening
- •Participant had any current medical condition that, in the opinion of the Investigator, may have compromised their safety or compliance, precluded successful conduct of the study, or interfered with interpretation of the results
研究组 & 干预措施
Cohort 1: ALXN1830
SYNT001 Dose 1
干预措施: ALXN1830 (Drug)
Cohort 2: ALXN1830
SYNT001 Dose 2
干预措施: ALXN1830 (Drug)
结局指标
主要结局
Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
时间窗: Day 0 (after first dose) through Day 112
A TEAE was defined as any adverse event that starts on or after the first dose of study drug or occurs prior to the first dose and worsens in severity on or after the first dose of study drug, during the Treatment Period and Follow-up Period. A TEAE was considered "serious" if, in the view of either the investigator or sponsor, it resulted in any of the following outcomes: death, life-threatening adverse drug event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, or an event that may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously listed outcomes. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. All serious TEAEs were not considered related to the study drug.
次要结局
- Change From Baseline In Hemoglobin At Day 33(Baseline, Day 33)
- Maximum Serum Concentration (Cmax) On Day 0 And Day 28(Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose)
- Change From Baseline In Reticulocyte Count At Day 33(Baseline, Day 33)
- Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level(Day 112)