A Study of AEB1102 (Pegzilarginase) in Patients With Arginase 1 Deficiency

NCT03378531 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: CAEB1102-102A
• Study Type: interventional
• Target: 14
• Locations: 4 countries
• Sites: 7

Brief Summary

The purpose of this study is to determine the safety, tolerability, and efficacy of long-term intravenous and subcutaneous administration of pegzilarginase for the treatment of pediatric and adult patients with ARG1-D who complete Study CAEB1102-101A.

Conditions

Hyperargininemia; Arginase I Deficiency

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Treatment-related Adverse Events

  From start of study up to 262 weeks

• Number of Participants With Anti-pegzilarginase and Anti-PEG Antibodies

  From start of study up to 262 weeks

Secondary Outcomes (14)

• Plasma Arginine Levels

  From start of study up to 240 weeks

• Plasma GC Levels (Argininic Acid)

  From start of study up to 240 weeks

• Plasma GC Levels (Guanidinoacetic Acid)

  From start of study up to 240 weeks

• Plasma GC Levels (α-keto-δ-guanidinovaleric Acid)

  From start of study up to 240 weeks

• Plasma GC Levels (α-N-acetylarginine)

  From start of study up to 240 weeks

Study Arms (1)

pegzilarginase (AEB1102)

EXPERIMENTAL

Each patient may receive pegzilarginase administered IV or SC for up to 4 years.

Drug: AEB1102

Interventions

AEB1102 DRUG

modified human arginase I

Also known as: Co-ArgI-PEG, Pegzilarginase

pegzilarginase (AEB1102)

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (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

Sponsors & Collaborators

• Aeglea Biotherapeutics: collaborator
• Immedica Pharma AB: lead

Study Sites (7)

Stanford University School of Medicine

Stanford, California, 94305, United States

Location

University of Florida

Gainsville, 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: 33325055 DERIVED

MeSH Terms

Conditions

Hyperargininemia

Condition Hierarchy (Ancestors)

Urea Cycle Disorders, Inborn; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Vice President, Head of Global Integrated Evidence Generation
• Organization: Immedica Pharma AB

clinical@immedica.com

+46 8 533 39 500

Study Officials

• Mattias Rudebeck, PhD MSc BMedSc

  Immedica Pharma AB

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

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: September 24, 2026
• Results First Posted: September 24, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

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