A Study to Learn More About the Treatment of People With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Who Received Recombinant ADAMTS13 (rADAMTS13) as Part of the Early Access Program

Condition(s) studied

Investigational drug(s) / intervention(s)

No Intervention: This is a non-interventional study.

Study summary

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.

Eligibility

Primary outcome measure(s)

• Number of cTTP Acute and Subacute Episodes Before and During Treatment with rADAMTS13 — 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 — 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 — 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 — 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 — 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.
• Changes in Biochemical Measures (Serum creatinine) Before and During Treatment with rADAMTS13 — 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 — 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 Participants with cTTP Symptoms Before and During Treatment with rADAMTS13 — 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).

Trial sites (8)

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