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

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

Study Overview

• Status: Active, not recruiting
• Conditions: Lysosomal Disease
• Intervention / Treatment: Other: No intervention

• Study Type: Observational
• Enrollment (Estimated): 222

Contacts and Locations

Study Locations

• Mexico
  • Mexico City, Mexico, 05348
    • Takeda Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Pediatric and adult participants of all ages diagnosed with lysosomal diseases (Fabry disease, Gaucher disease, or Hunter syndrome) who are enrolled in the PSP and have consented to the use of their anonymized data for research purposes will be included in this study, regardless of whether they receive modular or hospital-based infusions.

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Participants with Lysosomal Diseases; All pediatric and adult participants diagnosed with lysosomal diseases (Fabry disease, Gaucher disease, or Hunter syndrome) who were enrolled in the Patient Support Program (PSP) and provided consent for the use of their anonymized data for research purposes will be included in the study. Retrospective data will be obtained from anonymized secondary sources, including the PSP database and the Global Safety Database (GSDB). Participants who received treatment through either modular (home-based) infusion or hospital-based infusion between August 2023 and July 2024 will be analyzed.

Other: No intervention; This is a non-interventional study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ratio of Treatment Adherence by Infusion Setting (Modular Infusion versus Hospital Infusion)	Treatment adherence will be analyzed according to the definition of the ratio between prescribed vials vs currently administered vials.	Up to 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants With Adverse Events (AE) Receiving Modular versus Hospital Infusion	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).	Up to 6 months

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

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Study record dates

Study Major Dates

• Study Start (Actual): December 11, 2025
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2026

Study Registration Dates

• First Submitted: March 20, 2026
• First Submitted That Met QC Criteria: March 20, 2026
• First Posted (Actual): March 27, 2026

Study Record Updates

• Last Update Posted (Actual): March 27, 2026
• Last Update Submitted That Met QC Criteria: March 20, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Drug Therapy
• Other Study ID Numbers: TAK-665-4005

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No