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

NCT07187440 · tracked via the Priya Life Science China tracker

Sponsor

Takeda

Phase

Observational

Started

2025-11-01

Last updated

2026-01-02

Condition(s) studied

Fabry Disease

Investigational drug(s) / intervention(s)

No intervention

No intervention: This is a non-interventional study

Study summary

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 aims are to learn about the change in heart and kidney function, impact on quality of life, how the treatment with Replagal® works for people with Fabry Disease, and how safe the treatment with Replagal® is in routine real-world settings.

Participants will receive with Replagal® per the routine treatment settings in China. No study-specific visits to the clinical are scheduled.

Eligibility

Sex

ALL

Min age

7 Years

Max age

—

Healthy volunteers

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.

Primary outcome measure(s)

Annualized Rate of Change in Left Ventricular Mass Index (LVMI) — Up to 18 months
The annualized rate of change in LVMI will be estimated using linear mixed effects model.
Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) — Up to 18 months

Trial sites (18)

Facility	City	Region	Status
The First Affiliated Hospital Of USTC (AnHui Provincial Hospital)	Hefei	Anhui	Not Yet Recruiting
Peking University First Hospital	Beijing	Beijing Municipality	Not Yet Recruiting
Peking University Third Hospital	Beijing	Beijing Municipality	Recruiting
Peking Union Medical College Hospital	Beijing	Beijing Municipality	Recruiting
Fuzhou University Provincial Affiliated Hospital	Fuzhou	Fujian	Recruiting
The Second Affiliated Hospital ZheJiang University School Of Medicine	Hangzhou	Guangdong	Not Yet Recruiting
ShanTou Central Hospital	Shantou	Guangdong	Recruiting
The University Of Hong Kong-ShenZhen Hospital	Shenzhen	Guangdong	Not Yet Recruiting
The Second Hospital of Hebei Medical University	Shijiazhuang	Hebei	Recruiting
The Second Affiliated Hospital of Harbin Medical University	Harbin	Heilongjiang	Not Yet Recruiting
Affiliated Drum Tower Hospital, Medical School of Nanjing University	Nanjing	Jiangsu	Recruiting
The First Affiliated Hospital With NanJing Medical University(JiangSu Province Hospital)	Nanjing	Jiangsu	Not Yet Recruiting
The First Affiliated Hospital Of Soochow University	Suzhou	Jiangsu	Recruiting
The Second Affiliated Hospital of Xi 'an Jiaotong University	Xi'an	Shaanxi	Recruiting
The First Affiliated Hospital of Xi 'an Jiaotong University	Xi'an	Shaanxi	Not Yet Recruiting
Second Hospital Of ShanXi Medical University	Taiyuan	Shanxi	Not Yet Recruiting
West China Hospital of Sichuan University	Chengdu	Sichuan	Not Yet Recruiting
TianJin Chest Hospital	Tianjin	Tianjin Municipality	Not Yet Recruiting

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Other trials for the same condition

Fabry Disease Registry & Pregnancy Sub-registry Recruiting

Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease Active, not recruiting

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease Recruiting

Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07187440 on ClinicalTrials.gov ↗ ← All trials in China