A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With … (NCT03096080) | Clinical Trial Compass
CompletedPhase 1
A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
United States10 participantsStarted 2017-08-24
Plain-language summary
This study evaluates safety and tolerability of a single ascending dose of a tesevatinib liquid formulation administered to pediatric subjects with ARPKD.
Who can participate
Age range5 Years – 12 Years
SexALL
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Inclusion criteria
✓. Biliary ductal ectasia on magnetic resonance cholangiography or biliary duct ectasia or dilation on ultrasound
✓. Absence of renal cysts and/or characteristic imaging findings in both parents
✓. Signs of congenital periportal hepatic fibrosis as indicated by the presence of hepatosplenomegaly and/or esophageal varices and/or coarse liver echogenicity on ultrasound
✓. Hepatic periportal fibrosis on liver biopsy
✓. Pathologic (biopsy or autopsy) or genetic diagnosis of ARPKD in a deceased sibling or a clinical diagnosis of ARPKD in a living affected sibling
✓. Platelets \> 120,000/mm3
✓. Hemoglobin \> 9 g/dL
✓. Total bilirubin ≤ 1.5 mg/dL
Exclusion criteria
✕. History of torsade de pointes, ventricular tachycardia or fibrillation, pathologic sinus bradycardia (\< 50 bpm), heart block (excluding first-degree block, being PR interval prolongation only), congenital long QT syndrome or new ST segment elevation or depression or new Q wave on ECG. Subjects with a history of atrial arrhythmias should be discussed with the Medical Monitor
✕. Family history of congenital long QT syndrome or unexplained sudden cardiac death
✕. History of congenital prolonged QT syndrome, New York Heart Association class III or IV congestive heart failure
What they're measuring
1
Evaluation of the safety and tolerability of a single dose tesevatinib liquid formulation in pediatric participants with ARPKD