Safety and Pharmacodynamics of Intravenous Cangrelor in Pediatric Patients with Congenital Heart Disease Undergoing Percutaneous Vascular Procedures

The primary objective is to assess the safety profile of the recommended intravenous dosing regimen of cangrelor in children from birth to under 18 years undergoing diagnostic or therapeutic percutaneous vascular procedures for congenital heart disease. Safety evaluation will focus on adverse events, bleeding complications, and laboratory safety markers, providing data essential for determining tolerability of this procedural platelet inhibitor in a pediatric population.

The trial population comprised male and female subjects from birth up to but not including 18 years of age who had a confirmed diagnosis of Congenital Heart Disease and were scheduled for a diagnostic or therapeutic percutaneous vascular procedure. Both genders were eligible, and the cohort was defined as a vulnerable patient group. Selection was based on principal inclusion criteria, including the presence of specific cardiac lesions such as patent ductus arteriosus, coarctation of the aorta, atrial or ventricular septal defects, and valvular stenosis, as well as compliance with contraception requirements for participants of childbearing potential. No specific dietary, physical activity, or other lifestyle restrictions were stipulated in the eligibility description. The sponsor did not provide information regarding the total number of participants.

The study is a prospective, open-label, non‑randomized, multicentre interventional trial (Phase IIIb) evaluating the safety and pharmacodynamics of Cangrelor in pediatric subjects with Congenital Heart Disease undergoing diagnostic and/or therapeutic percutaneous vascular procedures. After a screening (inclusion) visit to confirm eligibility, eligible participants receive a single intravenous infusion of the investigational product immediately before the scheduled procedure. Follow‑up assessments are performed on Day 1, Day 2, and at discharge (typically Day 4), encompassing vital signs, laboratory parameters, and monitoring for bleeding, respiratory events, and renal function; an end‑of‑study visit follows discharge to document any delayed adverse events. Participant involvement therefore extends approximately four days from screening through the end‑of‑study assessment. Early termination criteria include occurrence of major bleeding, transfusion requirement related to the study drug, severe respiratory distress, a ≥2‑fold increase in creatinine, or any serious adverse event attributed to the investigational product. The primary endpoint is the proportion of subjects experiencing major, clinically relevant non‑major, or minor bleeding within 72 hours after the start of infusion. Recruitment is planned from June 2026 to January 2028.

The investigational product is Kengrexal 50 mg powder for concentrate for solution for injection/infusion, containing the active substance cangrelor tetrasodium. It is supplied as a solution for injection/infusion for intravenous administration. The protocol specifies a dose of 0 µg/kg administered intravenously; the exact volume is prepared according to the weight‑based calculation and infused as a single bolus prior to the vascular procedure.

No placebo or active comparator is incorporated in this study; participants receive only the investigational medication in conjunction with standard procedural care as determined by the treating physician.

Dosing is performed immediately before the percutaneous vascular intervention. The preparation and administration are documented in the case report form, and infusion timing is recorded to ensure adherence to the recommended schedule. Compliance with the dosing protocol is monitored through real‑time observation by study staff and verification of infusion logs.