A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Pegzilarginase Weekly subcutaneous dosing of pegzilarginase plus individualized disease management for 52 weeks |
Drug: Pegzilarginase
Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated
Other Names:
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Outcome Measures
Primary Outcome Measures
- 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)
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Prior enrollment in pegzilarginase clinical studies, with completion of treatment or continued dosing as of the study termination date
-
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
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Negative pregnancy test within 7 days prior to dosing in childbearing subjects if they are sexually active
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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:
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Subjects with acute or ongoing hyperammonemia within 6 weeks of the first dose
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Subjects with medical conditions or underlying issues that, in the opinion of the Investigator, would preclude participation in the study
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Aeglea Biotherapeutics
Investigators
- Study Director: Josie Gayton, Aeglea BioTherapeutics, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CAEB1102-305