A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in Pediatric Patients with Solid Tumors Dependent on KIT or PDGFRA Signaling

Conditions

Solid Tumors Dependent on KIT or PDGFRA Signaling

Brief summary

Part 1. Determination of the Part 2 recommended dose based on dose-limiting toxicity (DLT)., Part 1. Incidence and severity of adverse events, Part 2.Objective response rate (ORR)

Detailed description

Part 1 ORR, Part 2. Incidence and severity of adverse events, Part 2. Palatability assessments as measured by the 5-point Hedonic scale, Part 1 and Part 2. DOR: median and estimated rate at 6 and 12 months, Part 1 and Part 2. PFS: median and estimated rate at 6 and 12 months, Part 1 and Part 2. DCR at 24 weeks, Part 1 and Part 2. Time to response, Part 1 and Part 2. Avapritinib PK parameters including maximum plasma concentration, time to maximum plasma drug concentration (Tmax), area under the plasma concentration-time curve from 0 to 24 hours (AUC0-24), terminal elimination half-life (T1/2), and plasma concentration-time profiles (AUCs), Part 1 and Part 2. Change from baseline in levels of KIT and PDGFRA mutant allele fractions in peripheral blood at selected time points.

Related papers

No related papers found yet.

Interventions

DRUGAvapritinib

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Part 1. Determination of the Part 2 recommended dose based on dose-limiting toxicity (DLT)., Part 1. Incidence and severity of adverse events, Part 2.Objective response rate (ORR)	—

Secondary

Measure	Time frame
Part 1 ORR, Part 2. Incidence and severity of adverse events, Part 2. Palatability assessments as measured by the 5-point Hedonic scale, Part 1 and Part 2. DOR: median and estimated rate at 6 and 12 months, Part 1 and Part 2. PFS: median and estimated rate at 6 and 12 months, Part 1 and Part 2. DCR at 24 weeks, Part 1 and Part 2. Time to response, Part 1 and Part 2. Avapritinib PK parameters including maximum plasma concentration, time to maximum plasma drug concentration (Tmax), area under the plasma concentration-time curve from 0 to 24 hours (AUC0-24), terminal elimination half-life (T1/2), and plasma concentration-time profiles (AUCs), Part 1 and Part 2. Change from baseline in levels of KIT and PDGFRA mutant allele fractions in peripheral blood at selected time points.	—

Measure

Time frame

Part 1 ORR, Part 2. Incidence and severity of adverse events, Part 2. Palatability assessments as measured by the 5-point Hedonic scale, Part 1 and Part 2. DOR: median and estimated rate at 6 and 12 months, Part 1 and Part 2. PFS: median and estimated rate at 6 and 12 months, Part 1 and Part 2. DCR at 24 weeks, Part 1 and Part 2. Time to response, Part 1 and Part 2. Avapritinib PK parameters including maximum plasma concentration, time to maximum plasma drug concentration (Tmax), area under the plasma concentration-time curve from 0 to 24 hours (AUC0-24), terminal elimination half-life (T1/2), and plasma concentration-time profiles (AUCs), Part 1 and Part 2. Change from baseline in levels of KIT and PDGFRA mutant allele fractions in peripheral blood at selected time points.

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Countries

Austria, France, Germany, Italy

Outcome results

None listed