AI Cure is a research tool showing open data. Not medical advice. Discuss with your doctor. Terms

← Trials
🧪

A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease

Recruitingn/aAges 7 Years - anyAllTakeda
💬

In plain words

What the trial is, and whether it could suit you

Fabry Disease is a rare blood disorder that some people are born with. People with Fabry disease have low levels of an enzyme called alpha-galactosidase A. This enzyme helps to cut down fat-like substances. Without alpha-galactosidase A, large forms of these substances build up and clot in blood vessels. Over time, this can affect vital organs (especially the heart, kidneys, and brain) causing serious health problems with advancing age. Agalsidase alfa (Replagal®) is a human enzyme made in the laboratory and may provide higher levels of alpha-galactosidase A. Replagal® works the same way as natural alpha-galactosidase A does. The main aim of this study is to learn more about the treatment with Replagal® in Chinese children and adults with Fabry disease. The study aims to assess the heart and kidney function in people with Fabry disease who are routinely treated with Replagal®. Other aim

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
Inclusion Criteria Participants who meet all of the following criteria are eligible for this study: * Participant is greater than or equal to (\>= 7) years old. * Participant with confirmed diagnosis of Fabry disease (by investigator). * Participant never received or has received ERT (agalsidase alfa or agalsidase beta) within 12 weeks at most prior to enrolment. * Participant who will receive ERT with agalsidase alfa in routine clinical practice settings. * For \>= 18 years old, participant should sign the informed consent form (ICF); for 8-17 years old, participant and his (her) parents/ legally authorized representative (LAR) should both sign the ICF; for \< 8 years old, participant will give assent and his (her) parents/legally authorized representative should sign the ICF accordingly. Exclusion Criteria * Participant will be excluded if they have documented New York Heart Association (NYHA) functional Class IV heart failure symptoms (Implantable cardioverter-defibrillator \[ICD\] implanted excluded), third-degree atrioventricular block (ICD implanted excluded), acute myocardial infarction within the last 3 months and severe stroke (NIH Stroke Scale \[NIHSS\] \>= to 21). * Participant has enrolled in Fabry disease interventional clinical trial currently. * Other situations that the investigator considers not suitable for participation in this study.
💊

What it tests

  • No intervention
📍

Where

18 sites
The First Affiliated Hospital Of USTC (AnHui Provincial Hospital)
Hefei, China
Peking University First Hospital
Beijing, China
Peking University Third Hospital
Beijing, China
Peking Union Medical College Hospital
Beijing, China
Fuzhou University Provincial Affiliated Hospital
Fuzhou, China
The Second Affiliated Hospital ZheJiang University School Of Medicine
Hangzhou, China
ShanTou Central Hospital
Shantou, China
The University Of Hong Kong-ShenZhen Hospital
Shenzhen, China

…and 10 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
🛡️

AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it — not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use · Privacy