Evaluation of the Safety of Loargys Arginine Test System in Loargys-treated Patients
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Loargys Arginine Test System.
- Who it may be relevant to
- Registry conditions: ARG1 Deficiency. Basic parameters: 0 years — 100 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Multicenter, Prospective Study Assessing the Safety of Loargys Arginine Test System for Determining Arginine in Loargys-treated Patients
Overview
Arginase 1 deficiency (ARG1-D) is a rare condition in which the body cannot properly break down a substance called arginine. This leads to high levels of arginine and related substances in the blood, which can cause serious health problems and reduce quality of life. Loargys is a new treatment designed to lower arginine levels in people with ARG1-D. It works by providing a modified version of a natural enzyme that breaks down arginine in the blood. The dose of Loargys is adjusted for each patient based on their arginine levels. Because Loargys continues to break down arginine even after a blood sample has been taken, to ensure accurate results, a special substance (called nor-NOHA) is added to the blood collection tubes to stop this process after the sample is taken. The arginine levels are then measured using a standard laboratory test. The Loargys Arginine Test System used in this study includes both the special blood collection tubes and the laboratory test used to measure arginine. The purpose of this study is to evaluate the safety of this test system when used to measure arginine levels in patients receiving Loargys.
Interventions
- Device Loargys Arginine Test System
Loargys Arginine Test System
Primary outcome measures
- Incidence of AEs related to the Loargys Arginine Test System [Time frame: Through study completion, up to 21 months]
Eligibility criteria
Inclusion criteria
- Participant receiving treatment with commercialized Loargys for ARG1-D
- Participant is willing to sign the Informed Consent Form
Exclusion criteria
- None
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Other
Study locations
United States · 1 center
- Rare Disease Research — Atlanta
Identifiers
NCT: NCT07573059 · IMM-PEG-005