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

Primary Purpose

Arginase I Deficiency, Hyperargininemia

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

AEB1102

About this trial

This is an interventional treatment trial for Arginase I Deficiency

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Complete treatment in Study CAEB1102-101A without experiencing any clinically significant adverse event or other unmanageable drug toxicity that would preclude continued dosing
Confirmation by the Investigator and the Sponsor determine that it is acceptable for the patient to continue dosing with AEB1102
If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment
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
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

Sites / Locations

Stanford University School of Medicine
University of Florida
Icahn School of Medicine at Mount Sinai
UTSW
The Hospital for Sick Children
Centro Hospitalar S. Joao
Great Ormond Street Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

AEB1102

Arm Description

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

Outcomes

Primary Outcome Measures

Incidence of treatment-related adverse events

Secondary Outcome Measures

Cmax Cmin

Full Information

NCT ID

NCT03378531

First Posted

December 11, 2017

Last Updated

July 25, 2023

Sponsor

Aeglea Biotherapeutics

1. Study Identification

Unique Protocol Identification Number

NCT03378531

Brief Title

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

Acronym

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

Study Type

Interventional

2. Study Status

Record Verification Date

July 2023

Overall Recruitment Status

Completed

Study Start Date

December 7, 2017 (Actual)

Primary Completion Date

December 15, 2022 (Actual)

Study Completion Date

December 15, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Aeglea Biotherapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Arginase I Deficiency, Hyperargininemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

14 (Actual)

8. Arms, Groups, and Interventions

Arm Title

AEB1102

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

AEB1102

Other Intervention Name(s)

Co-ArgI-PEG, Pegzilarginase

Intervention Description

modified human arginase I

Primary Outcome Measure Information:

Title

Incidence of treatment-related adverse events

Description

Incidence of treatment-related adverse events

Time Frame

up to 4 years

Secondary Outcome Measure Information:

Title

Cmax Cmin

Description

Cmax Cmin

Time Frame

up to 4 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Complete treatment in Study CAEB1102-101A without experiencing any clinically significant adverse event or other unmanageable drug toxicity that would preclude continued dosing Confirmation by the Investigator and the Sponsor determine that it is acceptable for the patient to continue dosing with AEB1102 If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment 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 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Cortney Caudill

Organizational Affiliation

Aeglea Biotherapeutics, Inc.

Official's Role

Study Director

Facility Information:

Facility Name

Stanford University School of Medicine

City

Stanford

State/Province

California

ZIP/Postal Code

94305

Country

United States

Facility Name

University of Florida

City

Gainesville

State/Province

Florida

ZIP/Postal Code

32610

Country

United States

Facility Name

Icahn School of Medicine at Mount Sinai

City

New York

State/Province

New York

ZIP/Postal Code

10029

Country

United States

Facility Name

UTSW

City

Dallas

State/Province

Texas

ZIP/Postal Code

75390

Country

United States

Facility Name

The Hospital for Sick Children

City

Toronto

State/Province

Ontario

ZIP/Postal Code

M5G 1X8

Country

Canada

Facility Name

Centro Hospitalar S. Joao

City

Porto

Country

Portugal

Facility Name

Great Ormond Street Hospital

City

London

Country

United Kingdom

12. IPD Sharing Statement

Citations:

PubMed Identifier

33325055

Citation

Diaz GA, Schulze A, McNutt MC, Leao-Teles E, Merritt JL 2nd, Enns GM, Batzios S, Bannick A, Zori RT, Sloan LS, Potts SL, Bubb G, Quinn AG. Clinical effect and safety profile of pegzilarginase in patients with arginase 1 deficiency. J Inherit Metab Dis. 2021 Jul;44(4):847-856. doi: 10.1002/jimd.12343. Epub 2021 Jan 26.

Results Reference

derived

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

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