Treatment and Management of Patients With Congenital Thrombotic Thrombocytopenic Purpura (cTTP): An International, Multi-center Retrospective Chart Review of Patients in the Early Access Program (EAP) Treated With Recombinant ADAMTS13 (rADAMTS13)
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 94
- 主要终点
- Changes in Biochemical Measures (Serum creatinine) Before and During Treatment with rADAMTS13
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 inherited changes in the ADAMTS13 gene that reduce the body's ability to produce the ADAMTS13 enzyme. ADAMTS13 normally cleaves ultra-large multimers of a protein called von Willebrand factor (VWF). In cTTP, low ADAMTS13 activity allows these ultra-large VWF multimers to build up and promote blood clot formation in small blood vessels. These clots can restrict blood flow to vital organs and lead to serious complications.
Recombinant ADAMTS13 (rADAMTS13) is a manufactured form of human ADAMTS13 designed to replace the missing enzyme and restore ADAMTS13 activity.
This study aims to describe the impact of cTTP on participants before and after treatment with rADAMTS13. It will also evaluate participants' health outcomes after treatment and describe treatment patterns before and after rADAMTS13, including whether treatment was used to prevent or treat TTP episodes, how often it was given, the amount received, and others. In addition, the study will describe pregnancies and outcomes for the mother and baby before and during treatment with rADAMTS13.
Only data already available in the medical records of the people who received rADAMTS13 through Takeda's early access program (EAP) for cTTP will be collected and reviewed in 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
- •for the rADAMTS13 EAP are:
- •Participants of any age can participate who have a confirmed diagnosis of severe congenital ADAMTS13 deficiency or cTTP.
- •Participants must be on preventative or prophylactic treatment for cTTP or must have had at least one TTP event in the past.
- •Participants must have no other treatment options available (this includes other clinical studies for cTTP).
- •The inclusion criteria for this retrospective chart review are:
- •Pediatric and adult participants (no age restrictions) with cTTP, treated with rADAMTS13 via the EAP, who received at least two administrations of rADAMTS13 and who have provided consent (or the legal guardians) to participate in this retrospective chart review.
- •As per local regulations, evidence of a personally signed (or signed by a legally acceptable representative) and dated informed consent form/informed assent form (ICF/IAF) indicating that the participant (or their legal guardian) has been informed of all pertinent aspects of the retrospective chart review or an approval to process data without informed consent granted by an institutional review board/independent ethics committee (IRB/IEC)) Participants included in the EAP who were/are transitioned to the commercially available product will have their data abstracted for the duration of their participation in the EAP as well as when they received the commercially available product until the end of chart abstraction.
- •There are no additional
排除标准
- •for this chart review.
研究组 & 干预措施
Participants with cTTP
Data of participants who have received rADAMTS13 for the treatment of cTTP will be collected retrospectively from electronic medical records (eMR) for before (up to 6 months) and after initiation of rADAMTS13 treatment.
Participant enrollment and data collection for this study is expected to be completed by the last quarter of 2026.
干预措施: No Intervention (Other)
结局指标
主要结局
Changes in Biochemical Measures (Serum creatinine) Before and During Treatment with rADAMTS13
时间窗: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Biochemical measures like serum creatinine will be reported.
Changes in Biomarker Measures Before and During Treatment with rADAMTS13
时间窗: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in biomarker measures like ADAMTS13 activity level, total ADAMTS13 neutralizing/binding antibodies will be reported.
Number of cTTP Acute and Subacute Episodes Before and During Treatment with rADAMTS13
时间窗: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Acute episodes will be defined as those with clinically overt features of thrombotic microangiopathy, including thrombocytopenia, microangiopathic hemolytic anemia, and/or organ dysfunction, often requiring urgent therapeutic intervention. Subacute or non-overt episodes will be defined as episodes with laboratory evidence of thrombocytopenia or hemolysis without significant clinical symptoms or organ involvements, often detected on routine monitoring.
Changes in Hematological Measures (Platelet Count) Before and During Treatment with rADAMTS13
时间窗: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in hematological measures like platelet count (thrombocytopenia) will be reported.
Changes in Hematological Measures (Microangiopathic Hemolytic Anemia [MAHA]) Before and During Treatment with rADAMTS13
时间窗: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in hematological measures like MAHA will be reported.
Changes in Biochemical Measures (Lactate Dehydrogenase [LDH]) Before and During Treatment with rADAMTS13
时间窗: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Biochemical measures like LDH will be reported.
Changes in Biochemical Measures (Proteinuria) Before and During Treatment with rADAMTS13
时间窗: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Biochemical measures like Proteinuria will be reported.
Number of Participants with cTTP Symptoms Before and During Treatment with rADAMTS13
时间窗: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Number of participants with cTTP symptoms like abdominal pain, fatigue/lethargy, fever, bruising/purpura, neurological symptoms/stroke episodes, renal signs, thrombocytopenia, upper respiratory tract infections, headache/migraine, dizziness, diarrhea, nausea will be assessed using participant's electronic medical record (eMR).
次要结局
- Peak Activity Level of ADAMTS13(Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration)
- Trough Activity Level of ADAMTS13(Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration)
- Time of Sample Collection Relative to Infusion(Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration)
- Duration of Treatment and EAP Discontinuation With rADAMTS13(Up to end of treatment (up to 18 months))
- Number of Reasons for Treatment and EAP Discontinuation Before and During Treatment with rADAMTS13(Up to end of treatment (up to 18 months))
- Number of Participants Showing Response as per Laboratory Assessments After Treatment with rADAMTS13(After rADAMTS13 administration)
- Number of Reasons for rADAMTS13 Early Access Request(Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration)
- Number of Treatment Initiation Characteristics at the First Dose of rADAMTS13(From first dose of rADAMTS13 until EAP discontinuation (up to 6 months))
- Number of Treatment Changes During rADAMTS13 Treatment(From first dose of rADAMTS13 until EAP discontinuation (up to 6 months))
- Number of Instances of Pregnancy and its Corresponding Outcome Before and During Treatment with rADAMTS13(Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration)
- Duration of Infusion During Treatment with rADAMTS13(From First dose of rADAMTS13 until EAP discontinuation (Up to 6 months))
- Number of Days Spent in Hospital(From first dose of rADAMTS13 until EAP discontinuation (Up to 6 months))
- Number of Treatment Related Reasons for Hospitalization(From first dose of rADAMTS13 until EAP discontinuation (Up to 6 months))