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

NCT07429942 | Not Yet Recruiting

• 1 other identifier: TAK-755-4008
• Study Type: observational
• Target: 94
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Thrombotic Thrombocytopenic Purpura (TTP)

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (8)

• 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.

  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.

  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.

  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.

  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.

  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.

  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.

  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).

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

Secondary Outcomes (13)

• 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)

• 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

Study Arms (1)

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.

Other: No Intervention

Interventions

No Intervention OTHER

This is a non-interventional study.

Participants with cTTP

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adult and pediatric participants treated with rADAMTS13 via the EAP at participating centers will be included in this study.

You may qualify if:

• 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).
• 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.

Sponsors & Collaborators

• Takeda: lead

Related Links

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MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Thrombophilia; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Central Study Contacts

Takeda Contact

CONTACT

+1-877-825-3327; medinfoUS@takeda.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 28, 2026
• First Posted: February 24, 2026
• Study Start (Estimated): June 30, 2026
• Primary Completion (Estimated): July 6, 2026
• Study Completion (Estimated): July 6, 2026
• Last Updated: May 29, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.