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Fabry DiseaseNCT07187440

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

An observational study of Fabry Disease, run by Takeda at 18 sites in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages 7 years and older
Conditions the study is for
Fabry Disease
How many people
200 participants
Where
18 sitesChina
Study dates
1 Nov 2025to 30 Nov 2028
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 7 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Study population: Participants with Fabry disease who receive ERT with agalsidase alfa in routine clinical practice settings in China.

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

18 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Takeda Contact contact

+1-877-825-3327

medinfoUS@takeda.com

18 sites in 1 country

China18 sites, 18 recruiting

  • Peking University First Hospital

    Beijing, Beijing Municipality, 100034

    Site Contact, 13681050902, kmmawei@sina.com

    Wei Ma

    Not yet recruiting

  • Peking University Third Hospital

    Beijing, Beijing Municipality, 100191

    Site Contact, 13501277176, chenbaoxia@163.com

    Baoxia Chen

    Recruiting

  • Peking Union Medical College Hospital

    Beijing, Beijing Municipality, 100730

    Site Contact, 13911667211, Shuyangzhang103@163.com

    Shuyang Zhang

    Recruiting

  • West China Hospital of Sichuan University

    Chengdu, Sichuan, 610041

    Site Contact, 18980601267, qzhang2000cn@163.com

    Qing Zhang

    Not yet recruiting

  • Fuzhou University Provincial Affiliated Hospital

    Fuzhou, Fujian, 350001

    Site Contact, 13509384950, ysguo1234@163.com

    Yansong Guo

    Recruiting

  • The Second Affiliated Hospital ZheJiang University School Of Medicine

    Hangzhou, Guangdong, 310009

    Site Contact, 13588035801, meixiangxiang@zju.edu.com

    Meixiang Xiang

    Not yet recruiting

  • The Second Affiliated Hospital of Harbin Medical University

    Harbin, Heilongjiang, 150086

    Site Contact, 15904608957, dxy_shennei@126.com

    Xuanyi Du

    Not yet recruiting

  • The First Affiliated Hospital Of USTC (AnHui Provincial Hospital)

    Hefei, Anhui, 230001

    Site Contact, 13705511311, 13705511311@139.com

    Kangyu Chen

    Not yet recruiting

  • Affiliated Drum Tower Hospital, Medical School of Nanjing University

    Nanjing, Jiangsu, 210008

    Site Contact, 18115131666, wanglianglyy@163.com

    Lian Wang

    Recruiting

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

    Nanjing, Jiangsu, 210029

    Site Contact, 13611573111, xinli3267@yeah.net

    Xinli Li

    Not yet recruiting

  • ShanTou Central Hospital

    Shantou, Guangdong, 515031

    Site Contact, 18971490590, wangyingst@163.com

    Ying Wang

    Recruiting

  • The University Of Hong Kong-ShenZhen Hospital

    Shenzhen, Guangdong, 518053

    Site Contact, 18307556290, liumy@hku-szh.org

    Mingya Liu

    Not yet recruiting

  • The Second Hospital of Hebei Medical University

    Shijiazhuang, Hebei, '050000

    Site Contact, 13323218861, 2574459696@qq.com

    Songyun Zhang

    Recruiting

  • The First Affiliated Hospital Of Soochow University

    Suzhou, Jiangsu, 215006

    Site Contact, 13962176725, 1575677620@qq.com

    Bingyuan Zhou

    Recruiting

  • Second Hospital Of ShanXi Medical University

    Taiyuan, Shanxi, '030001

    Site Contact, 13546355355, yangbxys@163.com

    Bin Yang

    Not yet recruiting

  • TianJin Chest Hospital

    Tianjin, Tianjin Municipality, 300222

    Site Contact, 13002221869, chlfjp@sina.com

    Jinping Feng

    Not yet recruiting

  • The Second Affiliated Hospital of Xi 'an Jiaotong University

    Xi'an, Shaanxi, 710004

    Site Contact, 13669131478, lemonwine@126.com

    Li Wang

    Recruiting

  • The First Affiliated Hospital of Xi 'an Jiaotong University

    Xi'an, Shaanxi, 710061

    Site Contact, 13759930580, Luqun00@163.com

    Qun Lu

    Not yet recruiting

Other trials in China

About this study

Official title: 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

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.

Conditions
Fabry Disease
Being tested
No intervention
Keywords
Drug Therapy
Medical subject headings
Fabry Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • No interventionOther

    This is a non-interventional study

Study groups in NCT07187440
Study groupRoleWhat they receive
All ParticipantsParticipants with Fabry disease will receive enzyme replacement therapy (ERT) with agalsidase alfa under routine clinical practice settings.
Receives: Other: No intervention

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Annualized Rate of Change in Left Ventricular Mass Index (LVMI)

    The annualized rate of change in LVMI will be estimated using linear mixed effects model.

    Measured: Up to 18 months

  2. Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR)

    Measured: Up to 18 months

Secondary outcomes (14)

  1. Annualized Rate of Change in Left Ventricular Posterior Wall Dimensions (LVPWD)

    Measured: Up to 18 months

  2. Annualized Rate of Change in Ejection Fraction (EF)

    Measured: Up to 18 months

  3. Change From Baseline Over Time in LVMI

    Measured: Baseline, up to 18 months

  4. Change From Baseline Over Time in LVPWD

    Measured: Baseline, up to 18 months

  5. Change From Baseline Over Time in EF

    Measured: Baseline, up to 18 months

  6. Annualized Rate of Change in Urinary Albumin to Creatinine Ratio (UACR)

    Measured: Up to 18 months

  7. Annualized Rate of Change in Urine Protein Creatine Ratio (UPCR)

    Measured: Up to 18 months

  8. Change From Baseline Over Time in eGFR

    Measured: Baseline, up to 18 months

  9. Change From Baseline Over Time in UACR

    Measured: Baseline, up to 18 months

  10. Change From Baseline Over Time in UPCR

    Measured: Baseline, up to 18 months

  11. Change From Baseline Over Time in 24-hour (h) Urine Protein

    Measured: Baseline, up to 18 months

  12. Change From Baseline Over Time in 36-Item Short Form Health Survey (SF-36)

    SF-36 is a questionnaire that evaluates a person's HRQOL. SF-36 includes 36 questions related to 8 health dimensions: physical functioning, role-physical (role limitations due to physical health problems), bodily pain, general health, vitality (energy/fatigue), social functioning, role-emotional (role limitations due to emotional problems), and mental health. Based on these 4 scales (physical functioning, role-physical, bodily pain, general health), the physical component summary (PCS) score is generated which ranges between 0 and 100, with higher scores indicating a better quality of life. A positive change from Baseline indicates improvement.

    Measured: Baseline, up to 18 months

  13. Change From Baseline Over Time in Mainz Severity Score Index (MSSI)

    MSSI is an instrument for quantifying the overall severity of the signs and symptoms of Fabry disease. The MSSI assigns scores based on the presence and severity of signs and symptoms in four areas: general, neurologic, cardiovascular, and renal. Each of the signs and symptoms is weighted in accordance with its relationship to morbidity.

    MSSI scoring ranges from 0 (healthy) to 76 (maximum severity), and it is divided into severity bands of mild (<20), moderate (20-40), and severe (>40) affliction. Higher score indicates more severity.

    Measured: Baseline, up to 18 months

  14. Number of Participants With Clinically Significant Adverse Events (AEs) With Agalsidase Alfa Treatment

    Measured: Up to 18 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Sep 2025
  2. Study start1 Nov 2025
  3. Primary completion30 Nov 2028 (estimated)
  4. Study completion30 Nov 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
200 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07187440
Sponsor's protocol ID
TAK-675-4013
Status last verified
Dec 2025
First submitted
18 Sep 2025
Last updated
2 Jan 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Questions about this trial

What is this clinical trial studying?

An observational study of Fabry Disease, run by Takeda at 18 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 2 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 18 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Takeda Contact (+1-877-825-3327, medinfoUS@takeda.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Annualized Rate of Change in Left Ventricular Mass Index (LVMI) (time frame: Up to 18 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07187440.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jan 2026.

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