Cold Agglutinin Disease Real World Evidence Registry
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 429
- 试验地点
- 136
- 主要终点
- Sutimlimab cohort: number of blood cells transfusions
研究概览
简要总结
This is a multinational, multi-center, observational, prospective, longitudinal disease registry designed to collect data on participants with cold agglutinin disease (CAD) or cold agglutinin syndrome (CAS). Among them, a minimum of 30 patients with CAD treated with sutimlimab are expected to take part in the sutimlimab cohort study. Patients with CAD who have been enrolled in previous sutimlimab clinical trials (e.g., BIVV009-01/LTS16214 [NCT02502903,CAD patients], BIVV009-03/EFC16215 [NCT03347396], and BIVV009-04/EFC16216 [NCT03347422]) and who either completed or discontinued the corresponding clinical trial are eligible to participate in the registry.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
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
- •Patient aged ≥18 years
- •Patient able to understand the purpose of the study and who (or whose legally authorized representative) provided signed and dated informed consent and authorization to use protected health information in accordance with national and local subject privacy regulations
- •Patient with a diagnosis of CAD or CAS as per Investigator judgment based on the diagnosis criteria listed in study protocol
排除标准
- •Patient with mixed warm and cold autoimmune hemolytic anemia, or warm autoimmune hemolytic anemia
- •Patient actively participating in a CAD or CAS interventional clinical trial. After a patient completes participation in said trial, he/she may be eligible for enrollment in this registry.
研究组 & 干预措施
cold agglutinin syndrome (CAS)
Patient with a diagnosis of CAS as per investigator judgment based on the diagnosis criteria listed in study protocol
cold agglutinin disease (CAD)
Patient with a diagnosis of CAD as per investigator judgment based on the diagnosis criteria listed in study protocol. In addition, this group includes a cohort of CAD patients treated with sutimlimab
干预措施: Sutimlimab (Drug)
结局指标
主要结局
Sutimlimab cohort: number of blood cells transfusions
时间窗: Up to 6 years
Patterns of use of CAD and CAS treatments
时间窗: Up to 6 years
CAD or CAS treatments received before enrollment, at enrollment, and during the follow up period will be summarized. CAD or CAS symptoms and complications, autoimmune disorders or lymphoproliferative disorders, physician assessment of treatment status, and changes in laboratory results will be described by CAD or CAS treatments.
Cold Agglutinin Disease Symptoms and Impact Questionnaire (CAD-SIQ)
时间窗: Up to 6 years
Patterns of CAD and CAS disease characteristics
时间窗: Up to 6 years
CAD or CAS characteristics at diagnosis, enrollment, and during follow up (e.g., criteria used for diagnosis, symptoms, laboratory results) will be described.
Number of participants with health-resource utilization
时间窗: Up to 6 years
Health resource utilization may include hospitalizations (including length of stay), outpatient visits, emergency room visits, specific transfusion visits, nursing home/rehabilitation center stays, and home healthcare visits during registry follow-up period
Sutimlimab cohort: Sutimlimab treatment patterns
时间窗: Up to 6 years
Sutimlimab dosing information, reasons for dose adjustments, reasons for discontinuation will be described.
Number of participants with CAD or CAS complications
时间窗: Up to 6 years
Thromboembolic events (venous, arterial or cerebral), deaths related to CAD or CAS, other CAD/CAS complications (e.g., acute hemolytic crisis, hemolysis-related complications), and other comorbid conditions occurring before enrollment, at enrollment, and during follow up will be described.
Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue)
时间窗: Up to 6 years
36-Item Short Form Survey Instrument (SF-36) v2
时间窗: Up to 6 years
Sutimlimab cohort: Number of participants with adverse events, serious adverse events, adverse events of special interests (AESIs), other events of interests (e.g., Treatment emergent)
时间窗: Up to 6 years
Sutimlimab cohort: change from baseline in hemoglobin
时间窗: From baseline up to 6 years
Sutimlimab cohort: change from baseline in Lactate dehydrogenase (LDH)
时间窗: From baseline up to 6 years
Sutimlimab cohort: change from baseline in bilirubin
时间窗: From baseline up to 6 years
次要结局
未报告次要终点