Arginase I deficiency Hyperargininemia MedDRA version: 20.0 Level: PT Classification code 10062695 Term: Arginase deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Pegzilarginase
Product Code: AEB1102
Pharmaceutical Form: Solution for injection
INN or Proposed INN: PEGZILARGINASE
CAS Number: 1659310-95-8
Current Sponsor code: AEB1102
Other descript
Sponsors
Aeglea Biotherapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Prior enrollment in Studies 102A, 300A, or 301A, with completion of treatment or continued dosing as of the study termination date 2. Written informed consent by subject/parent/legal guardian, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol 3. Negative pregnancy test within 7 days prior to dosing in childbearing subjects if they are sexually active 4. If the subject (male or female) is engaging in sexual activity that could lead to pregnancy, must be surgically sterile, post-menopausal (female), or must agree to use a highly effective method of birth control during the study and for a minimum of 30 days after the last study drug administration. Highly effective methods of contraception include combined (estrogen and progestogen-containing) hormonal contraception associated with inhibition of ovulation, progesterone-only hormonal contraception associated with inhibition of ovulation, intrauterine device, or intrauterine hormone-releasing system. Are the trial subjects under 18? yes Number of subjects for this age range: 12 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 36 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subjects with acute or ongoing HA within 6 weeks of the first dose 2. Subjects with medical conditions or underlying issues that, in the opinion of the Investigator, would preclude participation in the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of long-term subcutaneous (SC) pegzilarginase in subjects with arginase 1 deficiency (ARG1-D);Secondary Objective: Not Applicable;Primary end point(s): Safety assessments that will be conducted as part of this study include the following: o Adverse events (AEs), including hypersensitivity reactions (HSRs), injection site reactions (ISRs), and hyperammonemic episodes (HAs) o Vital signs o Weight;Timepoint(s) of evaluation of this end point: Refer to protocol endpoint timepoints | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Not Applicable;Timepoint(s) of evaluation of this end point: Not Applicable | — |
Countries
Canada, France, Germany, Italy, Portugal, United Kingdom, United States
Contacts
Public ContactArg1-D Trial Information
Aeglea Biotherapeutics, Inc.
Outcome results
None listed