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

Phase 2

Completed

• Conditions: Hyperargininemia; Arginase I Deficiency
• Interventions: Drug: AEB1102

• Registration Number: NCT03378531
• Lead Sponsor: Aeglea Biotherapeutics

Brief 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.

Detailed 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.

Study Timeline

• Study Start: 2017-12-07
• Study First Submitted: 2017-12-11
• Study First Submitted QC: 2017-12-14
• Study First Posted: 2017-12-20
• Primary Completion: 2022-12-15
• Study Completion: 2022-12-15
• Status Verified: 2023-07
• Last Update Submitted: 2023-07-25
• Last Update Posted: 2023-07-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 14

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AEB1102	AEB1102	Each patient may receive AEB1102 administered IV for up to approximately 4 years.

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-related adverse events	up to 4 years	Incidence of treatment-related adverse events

Secondary Outcome Measures

Name	Time	Method
Cmax Cmin	up to 4 years	Cmax Cmin

Trial Locations

Locations (7)

Stanford University School of Medicine; 🇺🇸 Stanford, California, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

Icahn School of Medicine at Mount Sinai; 🇺🇸 New York, New York, United States

UTSW; 🇺🇸 Dallas, Texas, United States

The Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada

Centro Hospitalar S. Joao; 🇵🇹 Porto, Portugal

Great Ormond Street Hospital; 🇬🇧 London, United Kingdom