Agalsidase alfa (Replagal) enzyme replacement for Chinese children and adults with Fabry disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is an observational study that follows Chinese patients with Fabry disease who receive agalsidase alfa as part of routine clinical care. Participants aged 7 and older who are starting or continuing enzyme replacement therapy will have heart and kidney function, quality of life, and safety data collected over time. There is no experimental treatment assigned by the study; clinicians make treatment decisions and investigators record outcomes. The study is conducted at major hospitals in China and sponsored by Takeda to better understand real-world outcomes in the local population.

Why it matters

Who can join: the full rules

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.

Where it's running

• Peking University Third Hospital
  Beijing, Beijing Municipality, China Recruiting
• Peking Union Medical College Hospital
  Beijing, Beijing Municipality, China Recruiting
• Fuzhou University Provincial Affiliated Hospital
  Fuzhou, Fujian, China Recruiting
• ShanTou Central Hospital
  Shantou, Guangdong, China Recruiting
• The Second Hospital of Hebei Medical University
  Shijiazhuang, Hebei, China Recruiting
• Affiliated Drum Tower Hospital, Medical School of Nanjing University
  Nanjing, Jiangsu, China Recruiting

Study contacts

• Study coordinator: Takeda Contact
• Email: medinfoUS@takeda.com
• Phone: +1-877-825-3327

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Using advanced heart MRI to improve diagnosis and risk prediction for rare cardiomyopathies

  This project tests whether advanced cardiac MRI techniques can help diagnose and predict future risk for people with rare heart muscle diseases like Danon, Fabry, cardiac amyloidosis, Noonan syndrome, and cardiac sarcoidosis.

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• Quality of life for people 65+ with Fabry disease, treated or untreated

  This project will see if starting or continuing Fabry disease treatment changes quality of life over two and five years in people aged 65 and older.

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• Agalsidase alfa for heart inflammation in Fabry disease measured by PET‑CMR

  This study will test whether agalsidase alfa reduces heart inflammation in people with Fabry disease by comparing PET‑CMR scans before and after 12 months of treatment.

  Recruiting Yonsei University · Seoul · NCT07235709

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.