A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil
Mucopolysaccharidosis Type II in Brazil: A Retrospective Secondary Database Study (Informatics Department of Brazilian Health System - DATASUS)
2 other identifiers
observational
250
1 country
1
Brief Summary
The main aims of the study are to learn about the percentage of mucopolysaccharidosis type II (MPS II) in adults in Brazil as well as about the diagnosis process. No study medicines will be provided to participants in this study. The data available for participants diagnosed with MPS II in DATASUS (a database of the Informatics Department of Brazilian Health System) will be reviewed. No clinic visits will be required as part of participation in this study.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Dec 2021
Shorter than P25 for all trials
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 30, 2021
CompletedStudy Start
First participant enrolled
December 6, 2021
CompletedFirst Posted
Study publicly available on registry
December 13, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 15, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
February 15, 2022
CompletedMay 9, 2022
May 1, 2022
2 months
November 30, 2021
May 6, 2022
Conditions
Outcome Measures
Primary Outcomes (3)
Number of Participants Diagnosed With MPS II
Number of participants diagnosed with MPS II will be reported based on the retrospective data of participants collected from the database (DATASUS).
From January 1, 2008 to September 30, 2020
Number of Participants with Comorbidities Associated with MPS II
Comorbidities will be like respiratory infections, carpal tunnel syndrome and hernia. Number of participants with comorbidities associated with MPSII will be reported based on the retrospective data of participants collected from the database (DATASUS).
From January 1, 2008 to September 30, 2020
Age of MPS II Diagnosis
Age of MPS II diagnosis will be calculated from the date of birth and date of diagnosis. Retrospective data of participants will be collected from the database (DATASUS).
From January 1, 2008 to September 30, 2020
Secondary Outcomes (5)
Number of Health Care Professionals Who Diagnosed MPS II
From January 1, 2008 to September 30, 2020
Number of Health Care Professionals Consulted
From January 1, 2008 to September 30, 2020
Time to MPS II Diagnoses
From January 1, 2008 to September 30, 2020
Time Between MPS Diagnosis II and Start of Treatment
From January 1, 2008 to September 30, 2020
Number of Treatments Received Before and After MPS II Diagnosis
From January 1, 2008 to September 30, 2020
Study Arms (1)
Participants With MPS II
Retrospective data of participants diagnosed with MPS II will be collected from the database (DATASUS) from January 1st, 2008 to September 30th, 2020 in this observational study.
Interventions
Eligibility Criteria
Participants diagnosed with MPS II cases in Brazil.
You may qualify if:
- All MPS II cases of (International Classification of Diseases, 10th revision \[ICD-10\] E76.1) at any age reported in DATASUS from January 1st, 2008 to September 30th, 2020 will be included.
You may not qualify if:
- MPS II cases reported outside this period will be excluded.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Takedalead
Study Sites (1)
Takeda Distribuidora Ltda
São Paulo, 04794-000, Brazil
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 30, 2021
First Posted
December 13, 2021
Study Start
December 6, 2021
Primary Completion
February 15, 2022
Study Completion
February 15, 2022
Last Updated
May 9, 2022
Record last verified: 2022-05
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.
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.