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

NCT ID: NCT03378531

Last Updated: 2023-07-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

14 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-12-07

Study Completion Date

2022-12-15

Brief Summary

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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

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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.

Conditions

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Arginase I Deficiency Hyperargininemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AEB1102

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

Group Type EXPERIMENTAL

AEB1102

Intervention Type DRUG

modified human arginase I

Interventions

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AEB1102

modified human arginase I

Intervention Type DRUG

Other Intervention Names

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Co-ArgI-PEG Pegzilarginase

Eligibility Criteria

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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
Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Aeglea Biotherapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Cortney Caudill

Role: STUDY_DIRECTOR

Aeglea Biotherapeutics, Inc.

Locations

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Stanford University School of Medicine

Stanford, California, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status

UTSW

Dallas, Texas, United States

Site Status

The Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Centro Hospitalar S. Joao

Porto, , Portugal

Site Status

Great Ormond Street Hospital

London, , United Kingdom

Site Status

Countries

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United States Canada Portugal United Kingdom

References

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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.

Reference Type DERIVED
PMID: 33325055 (View on PubMed)

Other Identifiers

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CAEB1102-102A

Identifier Type: -

Identifier Source: org_study_id

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