NCT03455140

A Study Evaluating the Safety and Activity of Pegylated Recombinant Human Arginase (BCT-100)

A Phase I/II Study Evaluating the Safety and Activity of Pegylated Recombinant Human Arginase (BCT-100) in Relapsed/Refractory Cancers of Children and Young Adults

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 49 (actual)
• Sponsor: University of Birmingham · Academic / Other
• Sex: All
• Age: 1 Year – 25 Years
• Healthy volunteers: Not accepted

Summary

PARC is an international phase I/II trial evaluating the safety and activity of pegylated recombinant human arginase (BCT-100) in children and young people with relapsed/refractory leukaemia, neuroblastoma, sarcoma and high grade gliomas (brain cancers). Currently the outcomes for these patients are poor and the therapeutic options are limited with a significant toxicity burden. Therefore new treatments which work in different ways to standard chemotherapy are urgently needed. Research has shown that arginine (a nutrient) is important in the survival of cancer cells. BCT-100 is a drug which can deplete arginine levels and starve cancer cells - a completely new approach. BCT-100 has been tested in adults and shown to be active with almost no side-effects. This trial will test whether this dose of BCT-100 is also safe and active in children with relapsed/refractory leukaemia, neuroblastoma, sarcoma and high grade glioma. The trial will also study how BCT-100 is broken down in the body and look for new biological markers of treatment response. Up to 64 children with relapsed cancers will be recruited over 2 years.

Conditions

• Cancer
• Pediatric Solid Tumor
• Pediatric AML
• Pediatric ALL

Interventions

Type	Name	Description
DRUG	PEG- BCT-100	PEGylated recombinant human arginase 1

Timeline

• Start date: 2018-08-28
• Primary completion: 2022-07-22
• Completion: 2022-07-22
• First posted: 2018-03-06
• Last updated: 2022-09-02

Locations

14 sites across 3 countries: Australia, Netherlands, United Kingdom