craniosynostosis MedDRA version: 17.0 Level: PT Classification code 10049889 Term: Craniosynostosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: UGUROL
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Tranexamic Acid
Other descriptive name: TRANEXAMIC ACID
Concentration unit: mg/ml milligram(s)/millilitre
Concentrati
Sponsors
IRCCS Istituto Giannina Gaslini
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Infants and children, M or F, aged 3 months to 3 years, undergoing craniosynostosis repair, fronto-orbital advancement surgery and cranial remodeling surgery (i.e., total cavernal remodeling surgery); - Parents’/guardian written informed consent given before any study-related procedure not part of the subject’s normal medical care, with the understanding that consent may be withdrawn at any time without prejudice to child’s future medical care. Are the trial subjects under 18? yes Number of subjects for this age range: 60 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Preexisting hematological abnormality (defined as a positive history of bleeding disorder or a known diagnosis of a genetic or acquired bleeding disorder); - Preexisting coagulation defect (defined as PT, PTT or INR >1.5 times normal or a n pre-existing genetic or acquired coagulation defect); - Preexisting hepatic, renal, vascular, ocular and/or metabolic disorder; - History of acetylsalicylate administration within the last 14 days; - History of NSAIDs administration with 2 days of the scheduled surgery date.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the pharmacodynamics (PD), i.e. the clinical effectiveness (efficacy, tolerability and safety) of two dosing regimens of TXA in the target pediatric population. ;Secondary Objective: 1. To determine the population PK (popPK) of two dosing regimens. 2. To determine the PK/PD profiles of two TXA dosing regimens. 3. To evaluate the two TXA dosing regimens’ tolerability and safety profiles. 4. To explore the impact of PAI-1 gene polymorphism and levels on TXA clinical effects. ;Primary end point(s): The primary EP is the estimated volume of blood lost as calculated using the formula: ERCVlost = ERCVpreop + ERCVtransfused - ERCVpostop (where ERCV = Estimated Red Cell Volume). ;Timepoint(s) of evaluation of this end point: end of surgery | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Pop-PK concentration-time curves of two dosing regimens of TXA in the target population as influenced by demographic covariates such as age, body weight and renal function. 2. Concentration-effect relationships (PK/PD profiles) of two TXA dosing regimens to be evaluated and described and the appropriate statistical model to be fitted. 3. Proportion of patients with treatment-emergent adverse events (AEs) – i.e., thromboembolic events (vascular occlusive, stroke), neurologic events (seizures), serious AEs (SAEs), and laboratory abnormalities up to 90 days after surgery. 4. Effect of polymorphism of the PAI-1 gene and PAI-1 levels on TXA-mediated reduction in blood loss and transfusion requirement. ;Timepoint(s) of evaluation of this end point: end of surgery | — |
Countries
Italy, United States
Contacts
Public ContactEpidemiologia statistica e comitati
IRCCS Istituto Giannina Gaslini
Outcome results
None listed