NCT03378531

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Dec 2017

Longer than P75 for phase_2

Geographic Reach
4 countries

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 7, 2017

Completed
4 days until next milestone

First Submitted

Initial submission to the registry

December 11, 2017

Completed
9 days until next milestone

First Posted

Study publicly available on registry

December 20, 2017

Completed
5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 15, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 15, 2022

Completed
Last Updated

July 27, 2023

Status Verified

July 1, 2023

Enrollment Period

5 years

First QC Date

December 11, 2017

Last Update Submit

July 25, 2023

Conditions

Arginase I DeficiencyHyperargininemia

Outcome Measures

Primary Outcomes (1)

  • Incidence of treatment-related adverse events

    Incidence of treatment-related adverse events

    up to 4 years

Secondary Outcomes (1)

  • Cmax Cmin

    up to 4 years

Study Arms (1)

AEB1102

EXPERIMENTAL

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

Drug: AEB1102

Interventions

AEB1102DRUG

modified human arginase I

Also known as: Co-ArgI-PEG, Pegzilarginase
AEB1102

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Stanford University School of Medicine

Stanford, California, 94305, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

UTSW

Dallas, Texas, 75390, United States

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Centro Hospitalar S. Joao

Porto, Portugal

Location

Great Ormond Street Hospital

London, United Kingdom

Location

Related Publications (1)

  • 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.

    PMID: 33325055DERIVED

MeSH Terms

Conditions

Hyperargininemia

Condition Hierarchy (Ancestors)

Urea Cycle Disorders, InbornBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Cortney Caudill

    Aeglea Biotherapeutics, Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 11, 2017

First Posted

December 20, 2017

Study Start

December 7, 2017

Primary Completion

December 15, 2022

Study Completion

December 15, 2022

Last Updated

July 27, 2023

Record last verified: 2023-07

Locations

US(4)CA(1)PT(1)GB(1)