A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

The subject has had a previous partial or total nephrectomy.

The subject has any known genetic syndrome involving the kidney or liver other than ARPKD.

The subject has had clinically significant gastrointestinal bleeding during the 6 months prior to enrollment.

The subject has received any investigational therapy within 30 days prior to the first dose of study drug.

The subject has a history of pancreatitis, has known risk factors for pancreatitis, or baseline elevations in serum amylase or lipase.

The subject meets any of the following cardiac criteria:

1. 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

2. Family history of congenital long QT syndrome or unexplained sudden cardiac death

3. History of congenital prolonged QT syndrome, New York Heart Association class III or IV congestive heart failure

4. History of cardiac arrhythmias, stroke, or myocardial infarction

5. Has a cardiac pacemaker

The subject has an abnormal baseline audiogram.

The subject is taking or has taken any medication known to inhibit the cytochrome P450 (CYP) 3A4 isozyme or any drugs that are strong or moderate CYP3A4 inducers within 14 days prior to Day 1 of study drug.

The subject is taking or has taken any drugs associated with torsades de pointes or known to prolong the QTc interval, including anti-arrhythmic medications within 2 weeks prior to Day 1 of study drug.

The subject is receiving systemic anticoagulation.

The subject has an uncontrolled intercurrent illness that would limit compliance with study requirements.

The subject has an uncontrolled infection.

The subject is known to be positive for the human immunodeficiency virus or hepatitis B or C.

The subject is known to be immunocompromised.

The subject has any medical or surgical conditions that would interfere with gastrointestinal absorption of this oral agent.

The subject has received prior solid organ transplantation.

The subject, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.

The subject has an allergy or hypersensitivity to components of either the tesevatinib or the formulation.

The subject is aphakic.

The subject is pregnant or breast feeding.

A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

What this study is about

A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

What this study is about