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Active, Not Recruiting

NCT Number: NCT07494058

A Study of Home vs Hospital Treatment in People With Fabry, Gaucher or Hunter Conditions in Mexico

During the COVID-19 pandemic, home treatment for conditions such as Fabry, Gaucher or Hunter became important because it is easier for people to stick to their treatment if medicines that need to be given as infusion (called intravenous or IV treatment) can be given at home or somewhere close to home. Additionally, many of the hospitals that provide infusions are centralized in Mexico.

The main aim of the study is to find out whether people with Fabry, Gaucher or Hunter condition are more likely to continue and follow their treatment properly (called 'treatment adherence') when they receive it at home compared to when they receive it at a hospital. Other aims are to understand the factors that can influence treatment adherence, to learn about any regional differences in Mexico and to understand any medical problems with either treatment (home or hospital).

No treatment will be given during this study. Only already existing data will be reviewed during this study.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Lysosomal Disease

Sex eligibility

All sexes

Study type

Observational

Primary location

Takeda Site

Mexico City, 05348, Mexico

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participants who met the inclusion criteria of the "Modular infusion" program
  • Confirmed diagnosis of Fabry disease, Gaucher disease, or Hunter syndrome
  • Confirmatory diagnostic test
  • Referral to the PSP from the treating physician
  • Original PSP informed consent and
  • Privacy notice signed
  • Participants who met the inclusion criteria of the "Hospital infusion" program
  • Confirmed diagnosis of Fabry disease, Gaucher disease, or Hunter syndrome
  • Confirmatory diagnostic test
  • Referral to the PSP from the treating physician
  • Original PSP informed consent and
  • Privacy notice signed
  • Agreement that their anonymized information will be used for research purposes

Exclusion criteria

  • Participants who do not agree or withdraw his/her explicit consent for research purposes may have the right to withdraw; however, once their data has been integrated into the database, it is no longer identifiable by any participant in the research team.
  • Discontinuation of Participants

Treatment and study plan

No intervention

Other

This is a non-interventional study.

Primary outcomes

  1. Ratio of Treatment Adherence by Infusion Setting (Modular Infusion versus Hospital Infusion)

    Time frame: Up to 6 months

    Treatment adherence will be analyzed according to the definition of the ratio between prescribed vials vs currently administered vials.

Secondary outcomes

  1. Percentage of Participants With Adverse Events (AE) Receiving Modular versus Hospital Infusion

    Time frame: Up to 6 months

    AE is defined as any untoward medical occurrence in the participant notified to the pharmacovigilance unit, including Infusion-related reactions (observed within 24 hours of product administration).

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study title

Real-world Evidence of Modular Infusion Model in Lysosomal Disease: A Retrospective Observational Comparative Cohort Study From PSP (Patient Support Program) Data in Mexico

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Mar 27, 2026
Registry last updated
Mar 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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