Arginase I Deficiency, Hyperargininemia
Conditions
Keywords
ARG1-D
Brief summary
This is an open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.
Detailed description
Open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.
Interventions
DRUGPegzilarginase
Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated
Sponsors
Aeglea Biotherapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
1. Prior enrollment in pegzilarginase clinical studies, 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 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
Exclusion criteria
1. Subjects with acute or ongoing hyperammonemia 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 | Description |
|---|---|---|
| Treatment emergent adverse events | Baseline through week 54 | To evaluate the safety of long-term subcutaneous pegzilarginase in subjects with arginase 1 deficiency (ARG1-D) |
Countries
Canada
Outcome results
None listed