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

N/ANot Yet RecruitingNCT07429942

Takeda94 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Thrombotic Thrombocytopenic Purpura (TTP)

Interventions

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion criteria 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 exclusion criteria for this chart review.

Outcomes

Primary Outcomes

Number of cTTP Acute and Subacute Episodes Before and During Treatment with rADAMTS13

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.

Time frame: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

Changes in Hematological Measures (Platelet Count) Before and During Treatment with rADAMTS13

Changes in hematological measures like platelet count (thrombocytopenia) will be reported.

Time frame: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

Changes in Hematological Measures (Microangiopathic Hemolytic Anemia [MAHA]) Before and During Treatment with rADAMTS13

Changes in hematological measures like MAHA will be reported.

Time frame: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

Changes in Biochemical Measures (Lactate Dehydrogenase [LDH]) Before and During Treatment with rADAMTS13

Changes in Biochemical measures like LDH will be reported.

Time frame: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

Changes in Biochemical Measures (Proteinuria) Before and During Treatment with rADAMTS13

Changes in Biochemical measures like Proteinuria will be reported.

Time frame: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

Changes in Biochemical Measures (Serum creatinine) Before and During Treatment with rADAMTS13

Changes in Biochemical measures like serum creatinine will be reported.

Time frame: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

Changes in Biomarker Measures Before and During Treatment with rADAMTS13

Changes in biomarker measures like ADAMTS13 activity level, total ADAMTS13 neutralizing/binding antibodies will be reported.

Time frame: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

Number of Participants with cTTP Symptoms Before and During Treatment with rADAMTS13

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).

Time frame: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

Secondary Outcomes

Number of Reasons for rADAMTS13 Early Access Request

Number of reasons for early access request will be described.

Time frame: 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

Treatment initiation characteristics includes first rADAMTS13 treatment, participants weight at initiation of treatment, starting dose of rADAMTS13, other dose than specified in summary of product characteristics for prophylaxis or acute treatment, starting prophylaxis dose, frequency and duration will be reported.

Time frame: From first dose of rADAMTS13 until EAP discontinuation (up to 6 months)

Number of Treatment Changes During rADAMTS13 Treatment

Treatment changes during rADAMTS13 treatment (for each treatment episode) includes treatment purpose, treatment dates, location, doses and duration, any change(s) to dose, dosing frequency or duration, including dates and the reason(s), additional cTTP prophylaxis treatments will be reported.

Time frame: From first dose of rADAMTS13 until EAP discontinuation (up to 6 months)

Peak Activity Level of ADAMTS13

Peak ADAMTS13 activity level i.e., highest level post-infusion will be reported.

Time frame: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

Trough Activity Level of ADAMTS13

Trough ADAMTS13 activity level i.e., lowest level before next dose will be reported.

Time frame: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

Time of Sample Collection Relative to Infusion

Time of sample collection relative to infusion (1-hour pre infusion, 6 hours post infusion etc) will be reported.

Time frame: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

Number of Reasons for Treatment and EAP Discontinuation Before and During Treatment with rADAMTS13

Number of reasons for treatment and EAP discontinuation before and during treatment with rADAMTS13 will be reported.

Time frame: Up to end of treatment (up to 18 months)

Number of Participants Showing Response as per Laboratory Assessments After Treatment with rADAMTS13

Number of participants showing response as per laboratory assessments after treatment with radamts13 will be reported.

Time frame: After rADAMTS13 administration

Number of Instances of Pregnancy and its Corresponding Outcome Before and During Treatment with rADAMTS13

Number of instances of pregnancy and its corresponding Outcome, including pregnancies coinciding with exposure to rADAMTS13 will be reported.

Time frame: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration

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

Overview

Conditions

Interventions

Eligibility

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts