A Post-Authorization Safety Study (PASS) to Further Evaluate Real-World Safety in Patients With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 50
- 试验地点
- 7
- 主要终点
- Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions
研究概览
简要总结
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body's ability to produce a enzyme called ADAMTS13. This enzyme helps to cut down a larger form of protein called von Willebrand Factor (VWF). People with cTTP have low levels of ADAMTS13. Without ADAMTS13, large forms of vWF build up and cause blood clots in small blood vessels. These clots can block blood flow to vital organs, causing serious health problems. Adzynma is a human ADAMTS13 protein made in the laboratory. It works the same way as natural ADAMTS13 does and may provide higher levels of ADAMTS13.
The main aim of this study is to learn more about the risk of children and adults with cTTP treated with Adzynma developing antibodies that prevent Adzynma from working properly (called neutralizing antibodies) within 6 months after the first treatment and to understand the risk of allergic reactions within 7 days of the first treatment with Adzynma. Other aims are to better understand how safe treatment with Adzynma is over a longer period of time (called long-term safety) in children and adults with cTTP and to gather information about pregnancies and babies of women who have received Adzynma while pregnant.
Only data already available in the medical records of the people who received Adzynma for the treatment of cTTP in normal clinical practice will be reviewed and collected during this study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Is diagnosed with cTTP.
- •Had received commercially available Adzynma treatment for cTTP within the eligibility period of which there must be a recorded date and dose of at least one Adzynma administration in their medical chart.
- •Provides a signed informed consent form (informed consent form [ICF]; or assent and consent forms, if applicable), in accordance with local ethical and institutional requirements.
排除标准
- •Has a history or presence of a functional ADAMTS13 inhibitor (that is, neutralizing antibodies to ADAMTS13/rADAMTS13).
- •Has concurrent use of an investigational drug or is enrolled in another clinical trial at the time of index Adzynma infusion.
研究组 & 干预措施
All Participants
Data of participants who have received Adzynma for the treatment of cTTP will be collected retrospectively from medical records for approximately 5 years.
干预措施: No Intervention (Other)
结局指标
主要结局
Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions
时间窗: Up to 6 months following the index Adzynma infusion
Index infusion is defined as the first Adzynma infusion date recorded in the medical record using commercially available Adzynma (excluding doses received in clinical studies or early access program).
Number of Participants With Neutralizing Antibodies to ADAMTS13 Following the Index Adzynma Infusions
时间窗: Up to 6 months following the index Adzynma infusion
Number of participants with neutralizing antibodies to ADAMTS13 following the index Adzynma infusions will be reported.
次要结局
- Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma Infusion(Up to 6 months of follow-up time from the index Adzynma infusion)
- Number of Participants With Risk of Hypersensitivity Reactions After Each Administration of Adzynma Following the Index Infusion(Up to 7 days after each administration of Adzynma)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)(From start of the study drug administration up to 5.5 years)
- Gestational age at the Time of Infant Birth(At birth)
- Number of Participants With Spontaneous Abortion(Up to 5.5 years)
- Number of Participants With Stillbirth(Up to 5.5 years)
- Number of Participants With Induced Abortion(Up to 5.5 years)
- Number of Participants With Live Birth(Up to 5.5 years)
- Number of Participants With any Pregnancy Related Complications(Up to 5.5 years)
- Number of Participants Categorized According to Gestational age of Greater Than or Equal to (>=) 37 Gestational Weeks and Less Than (<) 37 Gestational Weeks at Birth(At birth)
- Number of Infants Categorized According to Year of Birth(Up to 5.5 years)
- Number of Infants With Normal Birth Weight(Up to 5.5 years)
- Number of Infants With Small for Gestational Age (SGA)(Up to 5.5 years)
- Number of Infants With Congenital Anomaly Detected at the Time of Birth(At birth)
- Number of Participants by Breastfeeding Status While Receiving Adzynma(Up to 5.5 years)
- Number of Infant Categorized by Growth and Development Outcomes(Up to 5.5 years)