NCT01956669

Pazopanib Paediatric Phase II Trial Children's Oncology Group (COG) in Solid Tumors

A Phase II Study of Pazopanib GW786034, NSC# 737754 in Children, Adolescents and Young Adults With Refractory Solid Tumors

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 57 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 1 Year – 18 Years
• Healthy volunteers: Not accepted

Summary

The study design was an open-label Phase II pediatric clinical study. The purpose of Study X2203 was to identify any efficacy signal in subjects with the disease subtypes under study, when treated with pazopanib monotherapy. Furthermore, it was to define the toxicities of pazopanib in children, as well as examine biological markers, e.g. cytokines and angiogenic factors, that could help further characterize any response of pazopanib in children. Pazopanib was administered as monotherapy in tablet and powder suspension formulations at daily doses of 450 mg/m2/dose or 225 mg/m2/dose, respectively. The first 6 enrolled subjects receiving oral suspension formulation were assessed for tolerability and extended PK sampling; and, only if pazopanib was tolerated, subsequent subjects were enrolled at the same starting dose with the suspension. Dose escalation was not permitted. For the tablet, a dosing nomogram was used based on the subject's BSA. Dose reduction was dependent upon the toxicity of pazopanib and disease status of the infants, toddlers, children, adolescents, and young adults. Subjects could be as young as 1 year-old infants to screen for enrollment. Subjects were assessed for initial response after 8 weeks of treatment prior to Cycle 3. A cycle was defined as 28 days of pazopanib treatment with no rest period between cycles. Treatment was administered continuously once daily. Treatment was to be discontinued if there was evidence of disease progression, unacceptable treatment-related toxicity, pregnancy. Histological classification was an important diagnostic inclusion in these subjects with a wide variety of refractory solid tumors, i.e. 7 different tumor types and each being a cohort.

Detailed description

The study design included a hierarchical 2-stage tumor response assessment with each cohort independent from one other. First, an initial 10 subjects were enrolled into each cohort. In the event of ≥ 1 response in any of these initial subjects, an additional 10 subjects may be enrolled in that cohort to proceed. In the event of ≥ 3 responses, the agent was considered effective. However after end of-stage 1, study enrollment stopped due to insufficient antitumor activity. Subjects who discontinued study treatment were followed for survival status. The study was to continue accruing data and thus, remained open for approximately 1 year from Last Subject Last Visit (LSLV).

Conditions

• Solid Tumours

Interventions

Type	Name	Description
DRUG	Pazopanib	Pazopanib was supplied as a series of aqueous film-coated tablets containing 200 mg (oval-shaped, white, packaged in bottles containing 34 tablets each), and 400 mg (oval-shaped, white, packaged in bottles containing 68 tablets each). Pazopanib Powder for Oral Suspension was a white to slightly colour powder supplied to the clinical sites in amber glass (United State Pharmacopeia (USP) Type III) bottles with child-resistant closures. Each bottle contains 5 g of pazopanib.

Timeline

• Start date: 2014-10-08
• Primary completion: 2019-11-05
• Completion: 2019-11-05
• First posted: 2013-10-08
• Last updated: 2020-08-12
• Results posted: 2020-08-12

Locations

31 sites across 7 countries: United States, Canada, Czechia, France, Hungary, Slovakia, Spain

Regulatory

• FDA-regulated drug study