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

NCT07494058 | Active Not Recruiting

• 1 other identifier: TAK-665-4005
• Study Type: observational
• Target: 222
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Lysosomal Disease

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

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

  Up to 6 months

Study Arms (1)

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

Interventions

No intervention OTHER

This is a non-interventional study.

Participants with Lysosomal Diseases

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Takeda: lead

Study Sites (1)

Takeda Site

Mexico City, 05348, Mexico

Location

Related Links

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Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 20, 2026
• First Posted: March 27, 2026
• Study Start: December 11, 2025
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.

Locations

ME (1)