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

NCT ID: NCT07187440

Last Updated: 2026-01-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

200 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-11-01

Study Completion Date

2028-11-30

Brief Summary

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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.

Detailed Description

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Conditions

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Fabry Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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All Participants

Participants with Fabry disease will receive enzyme replacement therapy (ERT) with agalsidase alfa under routine clinical practice settings.

No intervention

Intervention Type OTHER

This is a non-interventional study

Interventions

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No intervention

This is a non-interventional study

Intervention Type OTHER

Eligibility Criteria

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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.
Minimum Eligible Age

7 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Takeda

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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The First Affiliated Hospital Of USTC (AnHui Provincial Hospital)

Hefei, Anhui, China

Site Status NOT_YET_RECRUITING

Peking University First Hospital

Beijing, Beijing Municipality, China

Site Status NOT_YET_RECRUITING

Peking University Third Hospital

Beijing, Beijing Municipality, China

Site Status RECRUITING

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Site Status RECRUITING

Fuzhou University Provincial Affiliated Hospital

Fuzhou, Fujian, China

Site Status RECRUITING

The Second Affiliated Hospital ZheJiang University School Of Medicine

Hangzhou, Guangdong, China

Site Status NOT_YET_RECRUITING

ShanTou Central Hospital

Shantou, Guangdong, China

Site Status RECRUITING

The University Of Hong Kong-ShenZhen Hospital

Shenzhen, Guangdong, China

Site Status NOT_YET_RECRUITING

The Second Hospital of Hebei Medical University

Shijiazhuang, Hebei, China

Site Status RECRUITING

The Second Affiliated Hospital of Harbin Medical University

Harbin, Heilongjiang, China

Site Status NOT_YET_RECRUITING

Affiliated Drum Tower Hospital, Medical School of Nanjing University

Nanjing, Jiangsu, China

Site Status RECRUITING

The First Affiliated Hospital With NanJing Medical University(JiangSu Province Hospital)

Nanjing, Jiangsu, China

Site Status NOT_YET_RECRUITING

The First Affiliated Hospital Of Soochow University

Suzhou, Jiangsu, China

Site Status RECRUITING

The Second Affiliated Hospital of Xi 'an Jiaotong University

Xi'an, Shaanxi, China

Site Status RECRUITING

The First Affiliated Hospital of Xi 'an Jiaotong University

Xi'an, Shaanxi, China

Site Status NOT_YET_RECRUITING

Second Hospital Of ShanXi Medical University

Taiyuan, Shanxi, China

Site Status NOT_YET_RECRUITING

West China Hospital of Sichuan University

Chengdu, Sichuan, China

Site Status NOT_YET_RECRUITING

TianJin Chest Hospital

Tianjin, Tianjin Municipality, China

Site Status NOT_YET_RECRUITING

Countries

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China

Central Contacts

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Takeda Contact

Role: CONTACT

+1-877-825-3327

Facility Contacts

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Site Contact

Role: primary

13705511311

Site Contact

Role: primary

13681050902

Site Contact

Role: primary

13501277176

Site Contact

Role: primary

13911667211

Site Contact

Role: primary

13509384950

Site Contact

Role: primary

13588035801

Site Contact

Role: primary

18971490590

Site Contact

Role: primary

18307556290

Site Contact

Role: primary

13323218861

Site Contact

Role: primary

15904608957

Site Contact

Role: primary

18115131666

Site Contact

Role: primary

13611573111

Site Contact

Role: primary

13962176725

Site Contact

Role: primary

13669131478

Site Contact

Role: primary

13759930580

Site Contact

Role: primary

13546355355

Site Contact

Role: primary

18980601267

Site Contact

Role: primary

13002221869

Other Identifiers

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TAK-675-4013

Identifier Type: -

Identifier Source: org_study_id

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