NCT04337177

Flavored, Oral Irinotecan VAL-413 (Orotecan®) Given With Temozolomide for Treatment of Recurrent Pediatric Solid Tumors

Pilot Pharmacokinetic Study of VAL-413 (Orotecan®) in Patients With Recurrent Pediatric Solid Tumors

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Valent Technologies, LLC · Industry
• Sex: All
• Age: 1 Year – 30 Years
• Healthy volunteers: Not accepted

Summary

A pilot pharmacokinetic trial to determine the safety and efficacy of a flavored, orally administered irinotecan VAL-413 (Orotecan®) given with temozolomide for treatment of recurrent pediatric solid tumors including but not limited to neuroblastoma, rhabdomyosarcoma, Ewing sarcoma, hepatoblastoma and medulloblastoma

Detailed description

Up to 20 patients ≥ 1 year of age or ≤ 30 years of age with recurrent pediatric solid tumors will be enrolled. During the first cycle of treatment, each patient will receive 4 daily doses of VAL-413 (Orotecan®) and one daily dose of the intravenous preparation of irinotecan taken orally (IRN-IVPO), together with 5 days of concurrent temozolomide. During all subsequent cycles, only Orotecan® will be given with temozolomide in 5 day courses administered every 21 days as tolerated. The dosing regimen in this study will be Temozolomide at 100 mg/m2/day with Orotecan® at either 90 or 110mg/m2/day, administered orally for 5 consecutive days at the beginning of every 21-day cycle. A single dose of IRN-IVPO will be substituted at the same dosage as Orotecan® during Cycle 1. Up to 17 cycles of treatment may be administered on this study. Data collected from this study will allow for an assessment of Orotecan® safety and efficacy. Interval medical histories, targeted physical exams, complete blood counts, and other laboratory and safety assessments will be performed at Day 1 of each treatment cycle for all study subjects. At baseline and during study, disease status will be assessed by appropriate clinical and imaging evaluation (CT, MRI, or PET) and using Response Evaluation Criteria in Solid Tumors (RECIST), International Neuroblastoma Response Criteria (INRC) for patients with neuroblastoma, or the Children's Oncology Group Response Criteria for CNS tumors. In addition, a single-response taste survey will be conducted on Day 1 and Day 4 of the first cycle, which will allow patients to evaluate the taste of Orotecan®. Serum samples will be collected at various time points on Days 1 and 4 during Cycle 1 to characterize and compare the pharmacokinetic profiles of Orotecan® and conventional irinotecan given orally. Assessment of first-cycle toxicity will be used to identify the recommended phase II dose for Orotecan®. Toxicity will be evaluated and documented using NCI CTCAE guidelines. The recommended Phase II dose will be identified as the highest dose at which no more than 1 of 6 patients experiences a first cycle dose limiting toxicity (DLT). Additional study subjects may be enrolled at the recommended Phase II dose to ensure balanced safety and pharmacokinetic data is obtained for at least 3 patients \< 12 years old and at least 3 patients \> 12 years old.

Conditions

• Solid Tumors
• Neuroblastoma
• Rhabdomyosarcoma
• Ewing Sarcoma
• Hepatoblastoma
• Medulloblastoma

Interventions

Type	Name	Description
DRUG	VAL-413	a flavored preparation of orally administered irinotecan
DRUG	Temozolomide	alkylating oral chemotherapy agent used to treatment brain cancers

Timeline

• Start date: 2021-10-25
• Primary completion: 2026-06-01
• Completion: 2026-06-01
• First posted: 2020-04-07
• Last updated: 2025-09-23

Locations

8 sites across 1 country: United States

Regulatory

• FDA-regulated drug study