Lysosomal DiseaseNCT07494058
A Study of Home vs Hospital Treatment in People With Fabry, Gaucher or Hunter Conditions in Mexico
An observational study of Lysosomal Disease, run by Takeda at 1 site in Mexico.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Lysosomal Disease
- Type of treatment
- Injection or infusion
- How many people
- 232 participants
- Where
- 1 siteMexico
- Study dates
- 11 Dec 2025to 3 Jul 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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
Show all 13Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official 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
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
- Being tested
- No intervention
- Keywords
- Drug Therapy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
This is a non-interventional study.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 6 months
Secondary outcomes (1)
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).
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2026
- Study start11 Dec 2025
- Primary completion3 Jul 2026
- Study completion3 Jul 2026
Why it stopped: Study was terminated early due to structural and methodological limitations identified in the primary data sources impacting validity, completeness, and clinical robustness of the expected results
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lysosomal DiseaseStudies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 232 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07494058
- Sponsor's protocol ID
- TAK-665-4005
- Status last verified
- Sep 2026
- First submitted
- 20 Mar 2026
- Last updated
- 11 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT07494058
- Acronym / sponsor ID
- TAK-665-4005
- Conditions
- Lysosomal Disease
- Keywords
- Drug Therapy
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Study was terminated early due to structural and methodological limitations identified in the primary data sources impacting validity, completeness, and clinical robustness of the expected results
- Enrolment
- 232 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 11 Dec 2025 (actual)
- Primary completion
- 3 Jul 2026 (actual)
- Study completion
- 3 Jul 2026 (actual)
- First posted
- 27 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants with Lysosomal Diseases | – | Other: No interventionAll 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (13 listed)
- 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
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventionThis is a non-interventional study. | Other | Participants with Lysosomal Diseases |
Primary outcome
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.
1 secondary outcome
Percentage of Participants With Adverse Events (AE) Receiving Modular versus Hospital Infusion
Time frame: Up to 6 months
Results and safety
The study was stopped early (reason given: Study was terminated early due to structural and methodological limitations identified in the primary data sources impacting validity, completeness, and clinical robustness of the expected results). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2026.
- 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.Link provided by the sponsor.
- Click here for more information about this trial in easy-to-understand language.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Lysosomal Disease, run by Takeda at 1 site in Mexico.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 11 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Mexico. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Study was terminated early due to structural and methodological limitations identified in the primary data sources impacting validity, completeness, and clinical robustness of the expected results). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ratio of Treatment Adherence by Infusion Setting (Modular Infusion versus Hospital Infusion) (time frame: Up to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Sep 2026.