ClinicalTrials.gov record NCT07187440

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age7 years old and older
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a prospective, non-interventional study that follows Chinese children and people with Fabry Disease who receive agalsidase alfa (Replagal) in routine care. The study aims to describe heart and kidney measurements, quality of life, and safety while patients are treated as usual. No study-specific clinic visits are planned.

What it is studying. The study is testing how agalsidase alfa is used and what clinical outcomes occur in real-world Chinese patients with Fabry Disease. Key outcomes include changes over time in heart structure and function, kidney function and urine protein measures, patient-reported quality of life, disease severity scores, and the occurrence of adverse events, observed for up to 18 months.

What the researchers hope to learn. To describe clinical outcomes and safety of agalsidase alfa in routine practice among Chinese patients with Fabry Disease, including annualized changes in heart and kidney measures, quality of life, disease severity scores, and rates of clinically significant adverse events.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Other
  • No intervention

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is an observational study: participants receive agalsidase alfa as part of their routine care and the study does not assign treatment. No study-specific clinic visits are scheduled. The study team will collect routine medical information and test results over up to 18 months — including heart and kidney measurements, urine tests, quality-of-life questionnaires (e.g., SF-36), and records of any adverse events. Appropriate consent or assent (with parental/LAR consent where required) is needed to join.

How the study is structured. A prospective observational cohort: participants receive agalsidase alfa as part of routine clinical care (non-interventional). Study staff collect clinical measurements and questionnaires over time (up to about 18 months) during routine clinical care; the study does not assign treatments or add extra scheduled clinic visits.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Fabry disease

Clinical trials47 trials listed for Fabry diseaseEvery trial the registry lists for Fabry disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Fabry disease researchLatest monthly update: September 2026. Includes: SGLT2 outcome link. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Nov 1, 2025
Primary completion
Nov 30, 2028 (estimated)
Study completion
Nov 30, 2028 (estimated)
Target enrollment
200 participants (estimated)
Last updated on registry
Jan 2, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Takeda

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (18)

  • The First Affiliated Hospital Of USTC (AnHui Provincial Hospital)Not Yet Recruiting

    Hefei, Anhui, 230001 · China

  • Peking University First HospitalNot Yet Recruiting

    Beijing, Beijing Municipality, 100034 · China

  • Peking University Third HospitalRecruiting

    Beijing, Beijing Municipality, 100191 · China

  • Peking Union Medical College HospitalRecruiting

    Beijing, Beijing Municipality, 100730 · China

  • Fuzhou University Provincial Affiliated HospitalRecruiting

    Fuzhou, Fujian, 350001 · China

  • The Second Affiliated Hospital ZheJiang University School Of MedicineNot Yet Recruiting

    Hangzhou, Guangdong, 310009 · China

  • ShanTou Central HospitalRecruiting

    Shantou, Guangdong, 515031 · China

  • The University Of Hong Kong-ShenZhen HospitalNot Yet Recruiting

    Shenzhen, Guangdong, 518053 · China

  • The Second Hospital of Hebei Medical UniversityRecruiting

    Shijiazhuang, Hebei, '050000 · China

  • The Second Affiliated Hospital of Harbin Medical UniversityNot Yet Recruiting

    Harbin, Heilongjiang, 150086 · China

Terms used on this page · explanations by MedicalUpdates.ai

enrolment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
informed consent form
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
legally authorized representative
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
assent
A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
interventional
A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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