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

Aeglea BioTherapeutics

Status and phase: Completed; Phase 2

Conditions: Hyperargininemia; Arginase I Deficiency

Treatments: Drug: AEB1102

Study type: Interventional

Funder types: Industry

Identifiers

NCT03378531

CAEB1102-102A

Details and patient eligibility

About

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.

Full 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.

Enrollment: 14 patients

Sex: All

Ages: 2+ years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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14 participants in 1 patient group

AEB1102

Experimental group

Description:

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

Treatment:

Drug: AEB1102