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

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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03378531

Acronym

AEB1102

Enrollment

Registered

2017-12-20

Start date

2017-12-07

Completion date

2022-12-15

Last updated

2023-07-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Arginase I Deficiency, Hyperargininemia

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.

Interventions

DRUGAEB1102

modified human arginase I

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Incidence of treatment-related adverse events	up to 4 years	Incidence of treatment-related adverse events

Secondary

Measure	Time frame	Description
Cmax Cmin	up to 4 years	Cmax Cmin

Countries

Canada, Portugal, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026