Clinical trial NCT03378531: A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency
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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Arginase I Deficiency
Hyperargininemia
Lead sponsor: Aeglea Biotherapeutics
Sponsor class: INDUSTRY
First posted: 2017-12-20
Enrollment: 14
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Incidence of treatment-related adverse events
timeFrame: up to 4 years
Secondary outcomes:
measure: Cmax Cmin
timeFrame: up to 4 years
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University School of Medicine
state: California
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
city: Dallas
country: United States
facility: UTSW
state: Texas
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Porto
country: Portugal
facility: Centro Hospitalar S. Joao
city: London
country: United Kingdom
facility: Great Ormond Street Hospital
Study officials:
affiliation: Aeglea Biotherapeutics, Inc.
name: Cortney Caudill
role: STUDY_DIRECTOR
References:
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.