A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency

Hyperargininemia Clinical Trial

— AEB1102  

Official title:

An Open-label, Multicentre Extension Study to Evaluate the Long-Term Safety, Tolerability and Effects of Intravenous AEB1102 in Patients With Arginase I Deficiency Who Previously Received Treatment in Study CAEB1102-101A

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03378531
• Other study ID #: CAEB1102-102A
• Status: Completed
• Phase: Phase 2
• Start date: December 7, 2017
• Est. completion date: December 15, 2022

Study information

• Verified date: July 2023
• Source: Aeglea Biotherapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to investigate the long-term safety, tolerability, immunogenicity, pharmacokinetics and pharmacodynamics of intravenous AEB1102 in patients who complete Study CAEB1102-101A.

Description:

Purpose of this study is to investigate the long-term safety, tolerability, immunogenicity, pharmacokinetics and pharmacodynamics of intravenous AEB1102 in patients who complete Study CAEB1102-101A.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: December 15, 2022
• Est. primary completion date: December 15, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

Inclusion Criteria:

1. Complete treatment in Study CAEB1102-101A without experiencing any clinically significant adverse event or other unmanageable drug toxicity that would preclude continued dosing

2. Confirmation by the Investigator and the Sponsor determine that it is acceptable for the patient to continue dosing with AEB1102

3. If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment

4. If sexually active (male or female), must be surgically sterile, post-menopausal (female), or must agree to use a physician-approved method of birth control during the study and for a minimum of 30 days after the last study drug administration

5. Patient or legal guardian is able and willing to provide written informed consent and where required assent, and to comply with all requirements of study participation (including all study procedures and continuation of prescribed diet without modification), prior to any screening procedures

Exclusion Criteria:

1. Clinically significant concurrent disease, serious intercurrent illness, or other extenuating circumstances

Related Conditions & MeSH terms

• Arginase I Deficiency
• Hyperargininemia

Intervention

Drug:
• AEB1102
modified human arginase I

Locations

Country	Name	City	State
Canada	The Hospital for Sick Children	Toronto	Ontario
Portugal	Centro Hospitalar S. Joao	Porto
United Kingdom	Great Ormond Street Hospital	London
United States	UTSW	Dallas	Texas
United States	University of Florida	Gainesville	Florida
United States	Icahn School of Medicine at Mount Sinai	New York	New York
United States	Stanford University School of Medicine	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Aeglea Biotherapeutics

Countries where clinical trial is conducted

United States,  Canada,  Portugal,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-related adverse events	Incidence of treatment-related adverse events	up to 4 years
Secondary	Cmax Cmin	Cmax Cmin	up to 4 years