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

A Prospective, Multi-center, Observational Study to Evaluate the Clinical Outcomes of Agalsidase Alfa Enzyme Replacement Therapy Among Chinese Patients With Fabry Disease in Real-world Clinical Practice

This project will test whether agalsidase alfa (Replagal) helps heart and kidney health in Chinese children and adults with Fabry disease.

Quick facts

• Study type: Observational
• Enrollment: 200 (estimated)
• Ages: 7 Years and up
• Sex: All
• Sponsor: Takeda (Industry-sponsored)
• Locations: 18 sites (Hefei, Anhui and 17 other locations)
• Trial ID: NCT07187440 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are people in China aged 7 or older with a confirmed diagnosis of Fabry disease who will receive or continue agalsidase alfa (Replagal) as part of routine care and can provide informed consent or assent.

Not a fit: Patients with very advanced heart failure (NYHA Class IV), children under 7, or people who will not receive agalsidase alfa in routine care are unlikely to receive benefit from participating.

Why it matters

Potential benefit: If successful, the study could show that routine treatment with agalsidase alfa stabilizes or improves heart and kidney function and quality of life for Chinese patients with Fabry disease.

How similar studies have performed: Agalsidase alfa and other enzyme replacement therapies have been used globally and clinical studies have shown improvements in biochemical markers and some organ outcomes, although long-term benefits vary between reports.

Eligibility criteria

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 this trial is running

Hefei, Anhui and 17 other locations

• The First Affiliated Hospital Of USTC (AnHui Provincial Hospital) — Hefei, Anhui, China (Not_yet_recruiting)
• Peking University First Hospital — Beijing, Beijing Municipality, China (Not_yet_recruiting)
• 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)
• The Second Affiliated Hospital ZheJiang University School Of Medicine — Hangzhou, Guangdong, China (Not_yet_recruiting)
• ShanTou Central Hospital — Shantou, Guangdong, China (Recruiting)
• The University Of Hong Kong-ShenZhen Hospital — Shenzhen, Guangdong, China (Not_yet_recruiting)
• The Second Hospital of Hebei Medical University — Shijiazhuang, Hebei, China (Recruiting)
• The Second Affiliated Hospital of Harbin Medical University — Harbin, Heilongjiang, China (Not_yet_recruiting)
• Affiliated Drum Tower Hospital, Medical School of Nanjing University — Nanjing, Jiangsu, China (Recruiting)
• The First Affiliated Hospital With NanJing Medical University(JiangSu Province Hospital) — Nanjing, Jiangsu, China (Not_yet_recruiting)
• The First Affiliated Hospital Of Soochow University — Suzhou, Jiangsu, China (Recruiting)
• The Second Affiliated Hospital of Xi 'an Jiaotong University — Xi'an, Shaanxi, China (Recruiting)
• The First Affiliated Hospital of Xi 'an Jiaotong University — Xi'an, Shaanxi, China (Not_yet_recruiting)
• Second Hospital Of ShanXi Medical University — Taiyuan, Shanxi, China (Not_yet_recruiting)
• West China Hospital of Sichuan University — Chengdu, Sichuan, China (Not_yet_recruiting)
• TianJin Chest Hospital — Tianjin, Tianjin Municipality, China (Not_yet_recruiting)

Study contacts

• Study coordinator: Takeda Contact

Conditions Fabry Disease; Drug Therapy