A Study in the United Sates That Looks at the Safety and Effectiveness of Pradaxa Pellets in Children Aged 3 Months to Less Than 12 Years Who Need Treatment of a Blood Clot or Who Have Had a Blood Clot and Are at Risk of Developing Another Blood Clot

Safety and Effectiveness of Pradaxa Oral Pellet Formulation for Treatment of Acute Venous Thromboembolic Events (VTE) and/or for Risk Reduction of Recurrence of VTE in Pediatric Patients Aged 3 Months to Less Than 12 Years in a Real World Setting: a Prospective Non-interventional Study Conducted in the United States

The main research question of this study is to obtain further safety and effectiveness data on Pradaxa Pellets in children aged 3 months to less than 12 years in routine clinical practice setting.

Study Overview

• Status: Terminated
• Conditions: Venous Thromboembolism

• Study Type: Observational
• Enrollment (Actual): 5

Contacts and Locations

Study Locations

• United States
  • California
    • La Jolla, California, United States, 92093
      • University of California, San Diego
    • San Diego, California, United States, 92123
      • Rady Children's Hospital
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale University School of Medicine
  • Florida
    • St. Petersburg, Florida, United States, 33701
      • Johns Hopkins All Children's Hospital
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Indiana Hemophilia & Thrombrosis Center
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital
    • Dayton, Ohio, United States, 45404
      • Dayton Children's Hospital
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • MUSC (Medical university of South Carolina)
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University
  • Texas
    • Austin, Texas, United States, 78723
      • Dell Children's Ascension

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Pediatric patients aged 3 months to less than 12 years, who may be considered for anticoagulation with Pradaxa Pellets due to venous thromboembolic events (VTE), are usually treated in neonatology, pediatric general surgery, cardiac surgery or intensive care units. Pediatric patients with anticoagulation with Pradaxa Pellets for the prevention of recurrent VTE are usually evaluated by pediatric hematologists in pediatric hematology units. Any of these patients that are prescribed Pradaxa pellets may be considered for inclusion into this study.

Description

Inclusion Criteria:

Pediatric patients aged 3 months to less than 12 years at the time of Pradaxa Pellets initiation

• Written informed consent from parents/care givers and patient assent if age appropriate

• Initiation of Pradaxa Pellets administration either as initial or subsequent therapy:

  • Treatment of VTE
  • Treatment to reduce the risk of recurrence of VTE

Exclusion Criteria:

• Participation in any randomized clinical trial or use of investigational product, participation in any other observational study is not an exclusion
• Any contraindications to Pradaxa Pellets according to the US Prescribing Information.
• Previous participation in this study.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Pradaxa-treated patients; Pediatric patients with acute venous thromboembolic events (VTE) and/or at risk of recurrent VTE due to the presence of unresolved clinical risk factors received Pradaxa Pellets orally, either according to the prescribing label or off-label.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cumulative Incidence of Clinically Relevant Bleeding Events	Cumulative incidence of clinically relevant bleeding events was reported as the number of participants with clinically relevant bleeding events, defined as the composite of major bleeding events (MBE) and clinically relevant non-major (CRNM) bleeding events, according to recommendations from international thrombosis and hemostasis committees. MBE were defined as: fatal bleeding; clinically overt bleeding associated with a decrease in hemoglobin of at least 2 grams/deciliter in a 24-hour period; critical site bleeding; bleeding that required an intervention via invasive procedure; and overt bleeding for which a reversal agent was administered. CRNM bleeding was defined as: overt bleeding for which a blood product was administered and did not meet the criteria for major bleeding; bleeding that resulted in a medical or procedural intervention not meeting major bleeding criteria, including a medication change; and bleeding that resulted in hospitalization or an increased level of care.	From first Pradaxa Pellets exposure until its discontinuation plus 3 days of residual effect period, switch to other anticoagulation therapy, or planned observation time, whichever occurred first. Up to 373 days.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of Recurrent Venous Thromboembolic Event (VTE)	The occurrence of VTE is reported as the number of participants with recurrent VTE including both symptomatic and asymptomatic events. Symptomatic recurrent VTE is defined as radiologically-confirmed new venous thrombotic or embolic burden at least 7 days post-diagnosis of the index VTE, accompanied by signs and/or symptoms attributable to the new thromboembolism. Asymptomatic VTE was defined by new thrombotic/embolic burden as disclosed by comparison of end-of-treatment imaging versus baseline imaging of the vascular region involved by the index VTE.	From first Pradaxa Pellets exposure until its discontinuation plus 3 days of residual effect period, switch to other anticoagulation therapy, or planned observation time, whichever occurred first. Up to 373 days.
Mortality Related to Thrombotic or Thromboembolic Events	Number of participants who died with thrombotic or thromboembolic events.	From first Pradaxa Pellets exposure until its discontinuation plus 3 days of residual effect period, switch to other anticoagulation therapy, or planned observation time, whichever occurred first. Up to 373 days.
Occurrence of All Bleeding Events	The occurrence of all-bleeding events is reported as the number of participants experiencing any of the bleeding event described. All bleeding events were defined as the composite of major bleeding events (MBE), clinically relevant non-major (CRNM) bleeding events and minor bleeding events but not leading to hospitalization, increased level of inpatient care, or an intervention by the medical team. Minor bleeding events were defined as any overt or macroscopic evidence of bleeding that does not fulfil the criteria for major bleeding, CRNM bleeding, or important bleeding without intervention according to recommendations from international thrombosis and hemostasis committees.	From first Pradaxa Pellets exposure until its discontinuation plus 3 days of residual effect period, switch to other anticoagulation therapy, or planned observation time, whichever occurred first. Up to 373 days.
Occurrence of Post-thrombotic Syndrome (PTS)	Occurrence of post-thrombotic syndrome (PTS) is reported as the number of participants with PTS. The presence of PTS was evaluated by the Villalta Scale Modified for Children, the Manco-Johnson instrument, or other validated pediatric PTS instrument employed in routine clinical care, according to recommendations from international thrombosis and hemostasis committees.	From first Pradaxa Pellets exposure until its discontinuation plus 3 days of residual effect period, switch to other anticoagulation therapy, or planned observation time, whichever occurred first. Up to 373 days.
Incidence of Adverse Events (AEs)	Incidence of adverse events is reported as number of participants with any adverse event (AEs).	From first Pradaxa Pellets exposure until its discontinuation plus 3 days of residual effect period, switch to other anticoagulation therapy, or planned observation time, whichever occurred first. Up to 373 days.
Incidence of Serious Adverse Events (SAEs)	Incidence of serious adverse events is reported as number of participants with serious adverse event (SAEs).	From first Pradaxa Pellets exposure until its discontinuation plus 3 days of residual effect period, switch to other anticoagulation therapy, or planned observation time, whichever occurred first. Up to 373 days.
Thrombotic Burden at the End of Treatment	Number of participants with thrombotic burden at the end of the treatment period compared to baseline was quantified by image-based resolution status of the thrombus at the discretion of the treating physician, based on the type and location of the initial diagnosis of the thromboembolism. When appropriate, the same approach used for the baseline evaluations was utilized.	From first Pradaxa Pellets exposure until its discontinuation plus 3 days of residual effect period, switch to other anticoagulation therapy, or planned observation time, whichever occurred first. Up to 373 days.
Recurrence of Venous Thromboembolic Event (VTE) While on Treatment	Number of participants with new or recurrent VTE occurring at least 7 days after diagnosis of the VTE captured on the baseline VTE form (index VTE).	From first Pradaxa Pellets exposure until its discontinuation plus 3 days of residual effect period, switch to other anticoagulation therapy, or planned observation time, whichever occurred first. Up to 373 days.
Duration of Treatment With Dabigatran Etexilate	The median duration of the dabigatran etexilate (Pradaxa Pellets) treatment is reported.	From first Pradaxa Pellets exposure until its discontinuation. Up to 370 days.
Compliance With Dabigatran Etexilate Treatment	Number of participants complying with dabigatran etexilate (Pradaxa Pellets) treatment. Compliance was defined as not missing 0-1 treatment dose since the last visit, as evaluated at each time point. Unscheduled follow-up was defined as patient contact in between any scheduled study visit.	From first Pradaxa Pellets exposure until its discontinuation. At 6-week, 3-months, 6-month, and 12-month follow-up, and at unscheduled follow-up (up to 370 days).
Incidence of Adverse Events Leading to Drug Discontinuation	Incidence of adverse events leading to discontinuation of Pradaxa Pellets is reported as the number of participants.	From first Pradaxa Pellets exposure until its discontinuation plus 3 days of residual effect period, switch to other anticoagulation therapy, or planned observation time, whichever occurred first. Up to 373 days.

Collaborators and Investigators

• Sponsor: Boehringer Ingelheim
• Collaborators: Children's Hospital Acquired Thrombosis consortium

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): April 19, 2024
• Primary Completion (Actual): April 28, 2025
• Study Completion (Actual): April 28, 2025

Study Registration Dates

• First Submitted: July 21, 2023
• First Submitted That Met QC Criteria: July 21, 2023
• First Posted (Actual): August 1, 2023

Study Record Updates

• Last Update Posted (Actual): June 2, 2026
• Last Update Submitted That Met QC Criteria: June 1, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Embolism and Thrombosis; Thromboembolism; Venous Thromboembolism
• Other Study ID Numbers: 1160-0309

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Once the criteria in section "Time Frame" are fulfilled, researchers can use the following link https://www.clinicalstudies.boehringer-ingelheim.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement".

Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.

• IPD Sharing Time Frame: One year after the approval has been granted by major Regulatory Authorities and after the primary manuscript has been accepted for publication, or after termination of the development program.

IPD Sharing Access Criteria

For study documents - upon signing of a 'Document Sharing Agreement'.

For study data - 1. after the submission and approval of the research proposal (checks will be performed by the sponsor and/or the independent review panel, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a legal agreement.

• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No