NCT01498783

Phase I Study of 5-Fluorouracil in Children and Young Adults With Recurrent Ependymoma

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 26 (actual)
• Sponsor: St. Jude Children's Research Hospital · Academic / Other
• Sex: All
• Age: 1 Month – 21 Years
• Healthy volunteers: Not accepted

Summary

This is a phase I study to investigate the safety and pharmacokinetics of weekly 5-fluorouracil (5-FU) administered as a bolus dose in children and young adults with recurrent or refractory ependymoma. The results from this study will inform a subsequent phase II St. Jude investigator-initiated trial.

Detailed description

The initial 5-FU dosage will be 500 mg/m\^2 administered on day 1 of course 1. We plan to treat a maximum of 3 cohorts of research participants (dosage levels - 0, 1, and 2) with escalating doses of 5-FU. A cycle is defined as 42 days. The first 6 weeks of therapy will constitute the dose-limiting toxicity (DLT) evaluation period. Primary objective * To investigate the safety and pharmacokinetics (plasma and cerebrospinal fluid) of weekly bolus dose 5-FU in children and young adults with recurrent/refractory ependymoma * To study the safety of 500 mg/m\^2 weekly bolus dose 5-FU in less-heavily pre-treated children and young adults with recurrent/refractory ependymoma. Secondary objectives * To document and describe toxicities associated with 5-FU administered on a weekly bolus schedule * To document preliminary antitumor activity in participants with recurrent or refractory ependymoma treated with 5-FU * To assess the feasibility of measuring expression level of Thymidylate Synthetase (TYMS) in formalin fixed paraffin embedded (FFPE) tumor samples using the Quantigene assay * To evaluate the association between specific genetic polymorphisms (e.g., DPYD) and the pharmacokinetics of 5-FU

Conditions

• Central Nervous System Malignancies
• Ependymoma

Interventions

Type	Name	Description
DRUG	5-fluorouracil	5-fluorouracil, bolus dose of 500 mg/m\^2 given weekly for 4 weeks followed by a two week rest period equals one cycle (6 weeks). Therapy may continue for up to 16 cycles (about 2 years).

Timeline

• Start date: 2011-12-01
• Primary completion: 2014-05-01
• Completion: 2014-05-01
• First posted: 2011-12-23
• Last updated: 2016-02-02

Locations

1 site across 1 country: United States