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
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.Summary of Results
The study started in August 2024 and ended in June 2025. It was conducted in Austria, Portugal, and the United Kingdom.
The main goal of the study was to find out whether the medicine Loargys (pegzilarginase) is safe and can lower the levels of arginine in the blood of children with arginase 1 deficiency. Arginase 1 deficiency is a disease that causes arginine levels in the blood to become too high. The reason for this is that the enzyme called arginase, which usually breaks down arginine, is missing or not working properly in individuals with this disease.
The study was open for children under 2 years of age diagnosed with arginase 1 deficiency. Three children (2 boys and 1 girl) were treated in the study. At begin of the study, 1 child was 15 months old and the other 2 children were 23 months old.
Loargys is already used to treat arginase 1 deficiency in patients aged 2 years and older. The aim of this clinical study was to investigate if Loargys can be used effectively and safely in children younger than 2 years. The children were given Loargys once a week for 12 weeks by injection under the skin.
In this study, the blood of the children was tested for arginine at the beginning and end of the 12-week study treatment. After receiving Loargys once a week for 12 weeks, the children showed a meaningful reduction in their blood arginine levels.
The study doctor also assessed how well the children could start and complete certain movements such as lifting their head, crawling, standing and walking. This was done using a test called the Gross Motor Function Measure-66. After the study treatment, the children improved on this test by about 18%, which means their motor skills got better.
Individuals with arginase 1 deficiency sometimes also have high levels of ammonia in the blood. In this study, no child experienced episodes of high blood ammonia with symptoms that required hospital or emergency care. There were also no hypersensitivity reactions, and no cases where blood arginine levels dropped below the normal range for 14 days or longer.
Side effects are unwanted health problems that the doctors think were caused by Loargys. One child experienced mild redness of the skin at the injection site, which became better after 10 minutes without any treatment or hospital care, neither did it cause any lasting problems. Apart from this, no other side effects were reported.
This study helped researchers learn that Loargys is safe and may be effective in the treatment of arginase 1 deficiency in children younger than 2 years.
Please remember that this summary shows only the main results from one study. Generally, several studies are done to find out how well a medicine works and if it is safe.
Two European registry-based studies are currently being set up in collaboration with the E-IMD Registry Consortium where post-marketing data on the effectiveness and safety of Loargys in standard clinical setting is assessed.
The full study title is: A Phase 3 Open-Label Study of Safety, Pharmacokinetics, and Activity of Weekly Subcutaneous Pegzilarginase in Subjects <24 months old with Arginase 1 Deficiency
Study identification numbers:
Study no.: CAEB1102-301A
EU CT no.: 2024-510797-25
National Clinical Trials no.: NCT06582524
You can find more information about this study on the following websites:
https://clinicaltrials.gov/ (please enter NCT06582524 in the search field)
https://euclinicaltrials.eu/search-for-clinical-trials (please enter CAEB1102-301A or 2024-510797-25 in the search field)
If you have further questions about this study, please contact the sponsor of the study Immedica Pharma AB at clinical@immedica.com.
If your child took part in this study and you have further questions, please talk to the study doctor or staff at your study site.REC name
Wales REC 5
REC reference
24/WA/0141
Date of REC Opinion
7 Jun 2024
REC opinion
Further Information Favourable Opinion