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Not yet recruiting NCT07429942

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

— Observational Thrombotic Thrombocytopenic Purpura (TTP)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: No Intervention.
Who it may be relevant to
Registry conditions: Thrombotic Thrombocytopenic Purpura (TTP). Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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)

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.

Interventions

  • Other No Intervention
    This is a non-interventional study.

Primary outcome measures

  • Number of cTTP Acute and Subacute Episodes Before and During Treatment with rADAMTS13 [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 [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 [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 [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 [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 [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 [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 [Time frame: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration]
Secondary outcome measures (12)
  • Number of Reasons for rADAMTS13 Early Access Request [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 [Time frame: From first dose of rADAMTS13 until EAP discontinuation (up to 6 months)]
  • Number of Treatment Changes During rADAMTS13 Treatment [Time frame: From first dose of rADAMTS13 until EAP discontinuation (up to 6 months)]
  • Peak Activity Level of ADAMTS13 [Time frame: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration]
  • Trough Activity Level of ADAMTS13 [Time frame: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration]
  • Time of Sample Collection Relative to Infusion [Time frame: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration]
  • Duration of Treatment and EAP Discontinuation With rADAMTS13 [Time frame: Up to end of treatment (up to 18 months)]
  • Number of Reasons for Treatment and EAP Discontinuation Before and During Treatment with rADAMTS13 [Time frame: Up to end of treatment (up to 18 months)]
  • Number of Participants Showing Response as per Laboratory Assessments After Treatment with rADAMTS13 [Time frame: After rADAMTS13 administration]
  • Number of Instances of Pregnancy and its Corresponding Outcome Before and During Treatment with rADAMTS13 [Time frame: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration]
  • Duration of Infusion During Treatment with rADAMTS13 [Time frame: From First dose of rADAMTS13 until EAP discontinuation (Up to 6 months)]
  • Number of Days Spent in Hospital [Time frame: From first dose of rADAMTS13 until EAP discontinuation (Up to 6 months)]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07429942 · TAK-755-4008

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗