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

Официальное название

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

Обзор

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.

Вмешательства

• Другое No intervention
  This is a non-interventional study

Первичные конечные точки

• Annualized Rate of Change in Left Ventricular Mass Index (LVMI) [Срок оценки: Up to 18 months]
• Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) [Срок оценки: Up to 18 months]

Вторичные конечные точки (12)

• Annualized Rate of Change in Left Ventricular Posterior Wall Dimensions (LVPWD) [Срок оценки: Up to 18 months]
• Annualized Rate of Change in Ejection Fraction (EF) [Срок оценки: Up to 18 months]
• Change From Baseline Over Time in LVMI [Срок оценки: Baseline, up to 18 months]
• Change From Baseline Over Time in LVPWD [Срок оценки: Baseline, up to 18 months]
• Change From Baseline Over Time in EF [Срок оценки: Baseline, up to 18 months]
• Annualized Rate of Change in Urinary Albumin to Creatinine Ratio (UACR) [Срок оценки: Up to 18 months]
• Annualized Rate of Change in Urine Protein Creatine Ratio (UPCR) [Срок оценки: Up to 18 months]
• Change From Baseline Over Time in eGFR [Срок оценки: Baseline, up to 18 months]
• Change From Baseline Over Time in UACR [Срок оценки: Baseline, up to 18 months]
• Change From Baseline Over Time in UPCR [Срок оценки: Baseline, up to 18 months]
• Change From Baseline Over Time in 24-hour (h) Urine Protein [Срок оценки: Baseline, up to 18 months]
• Change From Baseline Over Time in 36-Item Short Form Health Survey (SF-36) [Срок оценки: Baseline, up to 18 months]

Критерии участия

Критерии включения

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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Китай · 18 центров

• The First Affiliated Hospital Of USTC (AnHui Provincial Hospital) — Хэфэй
• Peking University First Hospital — Пекин
• Peking University Third Hospital — Пекин
• Peking Union Medical College Hospital — Пекин
• Fuzhou University Provincial Affiliated Hospital — Фучжоу
• The Second Affiliated Hospital ZheJiang University School Of Medicine — Ханчжоу
• ShanTou Central Hospital — Shantou
• The University Of Hong Kong-ShenZhen Hospital — Шэньчжэнь
• … и ещё 10 центров

Идентификаторы

NCT: NCT07187440 · TAK-675-4013

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗