NCT03096080

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

A Phase 1, Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Kadmon Corporation, LLC · Industry
• Sex: All
• Age: 5 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

This study evaluates safety and tolerability of a single ascending dose of a tesevatinib liquid formulation administered to pediatric subjects with ARPKD.

Detailed description

This study is a phase 1 clinical trial focused on evaluating safety and tolerability of the Tesevatinib study drug, which is an oral solution at a 15mg/mL concentration. To determine safety of the tesevatinib liquid formulation in pediatric subjects (age 5-12) with ARPKD, all participants receive active study drug on Day 1 of the study enrollment. To evaluate plasma pharmacokinetics (PK) of the single dose of tesevatinib in the ARPKD pediatric subjects, the blood for PK sampling is drawn on Day 1, 2, and 3 of the study. Tesevatinib dosing will be followed by a PK and a 2-week safety evaluation. After the completion of the safety review subjects may continue onto the next dosing group at the discretion of the investigator and the medical monitor. There are three dosing arms in this study. Six participants will enroll into first dosing cohort (0.25mg/kg). Participants may be enrolled in two subsequent cohorts with increased dose (0.5mg/kg and 1.0mg/kg), if safety reporting is favorable. Medical history will be taken at Screening Visit. Echocardiogram will be performed at Screening and Day 14. Subjects will undergo audiology testing, as well as ocular monitoring at Screening and Day 14. Blood will be drawn for a panel of laboratory tests.

Conditions

• Polycystic Kidney, Autosomal Recessive

Interventions

Type	Name	Description
DRUG	Tesevatinib	One dose of the study drug in liquid form

Timeline

• Start date: 2017-08-24
• Primary completion: 2019-09-30
• Completion: 2019-09-30
• First posted: 2017-03-30
• Last updated: 2022-05-11

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study