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Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

NCT03921541 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2024-11-19

Study results available
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Summary

CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension; and a follow up visit for final safety assessments.

Conditions

  • Arginase I Deficiency
  • Hyperargininemia

Interventions

DRUG

Pegzilarginase

Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated

DRUG

Placebo

Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated

Sponsors & Collaborators

  • Aeglea Biotherapeutics

    lead INDUSTRY

Principal Investigators

  • Cortney Caudill · Aeglea BioTherapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-04-10
Primary Completion
2023-01-27
Completion
2023-01-27
FDA Drug
Yes

Countries

  • United States
  • Austria
  • Canada
  • France
  • Germany
  • Italy
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03921541 on ClinicalTrials.gov