CAEB1102-301A
Research type
Research Study
Full title
A Phase 3 Open-Label Study of Safety, Pharmacokinetics, and Activity of Weekly Subcutaneous Pegzilarginase in Subjects < 24 months old with Arginase 1 Deficiency
IRAS ID
1009597
Contact name
Mattias Rudebeck
Contact email
Sponsor organisation
Immedica Pharma AB
Eudract number
2024-510797-25
Clinicaltrials.gov Identifier
Research summary
This study is addressed to children below two years of age with arginase 1 deficiency (ARG1-D). ARG1-D is a rare inherited disease caused by low levels of the enzyme arginase 1, which breaks down the amino acid (the building blocks of protein) arginine. Low levels or no arginase can lead to increases in the level of arginine in the blood and tissues (hyperargininemia). The Sponsor develops a medicine called Loargys (pegzilarginase), an enzyme derived from human arginase 1 that has been modified for use as a medicine. The reason for this study is to find out about the safety of Loargys and its ability to lower arginine levels in patients younger than two years. In addition, it will be investigated how the body processes pegzilarginase. Loargys has been approved in the United Kingdom and European Union for patients aged 2 years and older but has not been studied in younger patients.
Over a treatment period of 12 weeks, study participants will receive once-weekly doses of Loargy as injection under the skin. In total, study participation will last approximately 24 weeks and include 15 scheduled study visits to the study center. During the study visits, blood tests (at 9 visits) and electrocardiograms (at 3 visits) will be performed.REC name
Wales REC 5
REC reference
24/WA/0141
Date of REC Opinion
7 Jun 2024
REC opinion
Further Information Favourable Opinion