drugset
drugset / Trial / NCT01528384

Ph 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient

NCT01528384 ↗

Phase 1 Completed 8 enrolled Polaris Group
NaSingle-groupOpen-labelTreatment

Summary

This is a phase 1 study of pegylated arginine deiminase (ADI-PEG 20) in pediatric patients who deficient in the expression of tumor argininosuccinate synthetase, and have failed prior treatments. The study hypothesis is that this drug will be safe in pediatric patients.

Timeline

Start
2011-12
Primary completion
2014-03
Completion
2014-03

Drugs

EvaluationDrugModalityDoseRoute
Subject Pegargiminase Protein / enzyme biologic 4.5 mg/m2 Intramuscular
Subject Pegargiminase Protein / enzyme biologic 9 mg/m2 Intramuscular
Subject Pegargiminase Protein / enzyme biologic 18 mg/m2 Intramuscular

Indications

argininosuccinic aciduria