← Back to Search

Enzyme Replacement Therapy

Pegzilarginase for Arginase I Deficiency

Phase 3

Waitlist Available

Research Sponsored by Aeglea Biotherapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 2, week 7, week 12, week 17, week 24

Awards & highlights

Study Summary

This trial will study whether pegzilarginase is safe and effective in patients with a rare disease called ARG1-D. The trial will have a screening period, a treatment period where some patients will receive the drug and some will receive a placebo, a long-term extension, and a follow-up visit.

Eligible Conditions

Arginase I Deficiency
Hyperargininemia

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 2, week 7, week 12, week 17, week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 2, week 7, week 12, week 17, week 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 2, week 7, week 12, week 17, week 24 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from baseline in plasma arginine concentration after 24 weeks of treatment

Secondary outcome measures

Change in ornithine and guanidino compounds

Evaluate immunogenicity of pegzilarginase

Evaluate safety of pegzilarginase

+9 more

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Pegzilarginase Long Term ExtensionExperimental Treatment1 Intervention

After completion of 24 weeks DB treatment, weekly IV infusions of pegzilarginase plus individualized disease management for an additional 150 weeks, with the option to receive treatment by SC after 8 weeks of the LTE study.

Group II: PegzilarginaseExperimental Treatment1 Intervention

Weekly IV infusions of pegzilarginase plus individualized disease management for 24 weeks

Group III: PlaceboPlacebo Group1 Intervention

Weekly IV infusions of placebo plus individualized disease management for 24 weeks

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pegzilarginase

2017

Completed Phase 3

~100

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Aeglea BiotherapeuticsLead Sponsor

7 Previous Clinical Trials

241 Total Patients Enrolled

3 Trials studying Arginase I Deficiency

33 Patients Enrolled for Arginase I Deficiency

Cortney CaudillStudy DirectorAeglea BioTherapeutics, Inc.

3 Previous Clinical Trials

30 Total Patients Enrolled

2 Trials studying Arginase I Deficiency

17 Patients Enrolled for Arginase I Deficiency

Josie GaytonStudy DirectorAeglea BioTherapeutics, Inc.

4 Previous Clinical Trials

101 Total Patients Enrolled

3 Trials studying Arginase I Deficiency

33 Patients Enrolled for Arginase I Deficiency

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any potential dangers to using Pegzilarginase?

"There is already some efficacy data for Pegzilarginase from Phase 3 trials, as well as multiple rounds of safety data, so it received a score of 3."

Answered by AI

How many people are involved in this clinical trial at most?

"Currently, this clinical trial is not recruiting patients. The last update to the study was on August 9th, 2022. There are, however, 4 other active studies related to argeninemia and Pegzilarginase."

Answered by AI

How many different places is this clinical trial being conducted?

"In addition to Stanford University School of Medicine in Stanford, California and Emory University in Atlanta, Georgia, this clinical trial is also taking place at Children's Hospital of Philadelphia in Philadelphia, Pennsylvania as well as 18 other locations."

Answered by AI

Are there other ongoing clinical trials that use Pegzilarginase?

"In 2017, the first clinical trials evaluating pegzilarginase's efficacy began at Centro Hospitalar S. Joao. As of now, a total of 18293 have been concluded with 2 more active studies underway. Most of these investigations are occurring in Stanford, California."

Answered by AI

Are new participants being accepted into this clinical trial?

"As of now, this trial is no longer recruiting patients. The study was initially posted on April 10th, 2019 and updated for the last time on August 9th, 2022. If you are looking for other trials, there are two studies for hyperargininemia and two more for Pegzilarginase that currently have open enrollment."

Answered by AI

What are the origins of this clinical trial?

"Since 2017, Pegzilarginase has undergone clinical trials. In the first study, which was sponsored by Aeglea Biotherapeutics and had 14 participants, it received Phase 2 drug approval in 2017. There are currently two ongoing trials for Pegzilarginase being conducted in 8 countries and 21 cities."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

2019. "Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03921541.