NCT01528384

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

A Phase 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient and Have Failed Prior Systemic Therapy

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Polaris Group · Industry
• Sex: All
• Age: 4 Years – 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This phase 1 study will use a classic 3 dose levels by 3 patients per dose level design. Once the maximum tolerated dose is reached, additional patients may be enrolled at that dose level.

Conditions

• Argininosuccinate Synthetase Deficient

Interventions

Type	Name	Description
DRUG	ADI-PEG 20	4.5, 9 and 18 mg/m2 dosage weekly dosing intramuscular until progressive disease

Timeline

• Start date: 2011-12-01
• Primary completion: 2014-03-01
• Completion: 2014-03-01
• First posted: 2012-02-08
• Last updated: 2014-04-09

Locations

1 site across 1 country: United States