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

Sponsor

Aeglea Biotherapeutics (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT03378531

Collaborator

(none)

Enrollment

Locations

Arm

65.8

Anticipated Duration (Months)

Patients Per Site

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Arginase I Deficiency Hyperargininemia	Drug: AEB1102	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

14 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Dec 7, 2017

Anticipated Primary Completion Date :

Jun 1, 2023

Anticipated Study Completion Date :

Jun 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: AEB1102 Each patient may receive AEB1102 administered IV for up to approximately 4 years.	Drug: AEB1102 modified human arginase I Other Names: Co-ArgI-PEG Pegzilarginase

Arm

Intervention/Treatment

Experimental: AEB1102

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

Drug: AEB1102

modified human arginase I

Other Names:

Co-ArgI-PEG

Pegzilarginase

Outcome Measures

Primary Outcome Measures

Incidence of treatment-related adverse events [up to 4 years]

Secondary Outcome Measures

Cmax Cmin [up to 4 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years and Older

Sexes Eligible for Study:

All

Accepts 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:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Stanford University School of Medicine	Stanford	California	United States	94305
2	University of Florida	Gainesville	Florida	United States	32610
3	Icahn School of Medicine at Mount Sinai	New York	New York	United States	10029
4	UTSW	Dallas	Texas	United States	75390
5	The Hospital for Sick Children	Toronto	Ontario	Canada	M5G 1X8
6	Centro Hospitalar S. Joao	Porto		Portugal
7	Great Ormond Street Hospital	London		United Kingdom

Sponsors and Collaborators

Aeglea Biotherapeutics

Investigators

Study Director: Josie Gayton, Aeglea Biotherapeutics, Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Aeglea Biotherapeutics

ClinicalTrials.gov Identifier:

NCT03378531

Other Study ID Numbers:

CAEB1102-102A

First Posted:

Dec 20, 2017

Last Update Posted:

Jul 25, 2022

Last Verified:

Jul 1, 2022

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.:

Additional relevant MeSH terms:

Hyperargininemia

Study Results

No Results Posted as of Jul 25, 2022