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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No intervention | Other | This is a non-interventional study. |
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| 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 |
| 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 |
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Inclusion Criteria
Participants who met the inclusion criteria of the "Modular infusion" program
Participants who met the inclusion criteria of the "Hospital infusion" program
Agreement that their anonymized information will be used for research purposes
Exclusion Criteria
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Takeda Site | Mexico City | 05348 | Mexico |
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| Label | URL |
|---|---|
| Click here to ask Takeda's chatbot for comprehensive and easy-to-understand information about clinical trials - even across products and indications - in your local language. | View source |
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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.
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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.
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