A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil

Completed

• Conditions: Mucopolysaccharidosis (MPS)
• Interventions: Other: No Intervention

• Registration Number: NCT05155488
• Lead Sponsor: Takeda

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-11-30
• Study First Submitted QC: 2021-11-30
• Study Start: 2021-12-06
• Study First Posted: 2021-12-13
• Primary Completion: 2022-02-15
• Study Completion: 2022-02-15
• Status Verified: 2022-05
• Last Update Submitted: 2022-05-06
• Last Update Posted: 2022-05-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 250

Inclusion Criteria

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.

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Exclusion Criteria

MPS II cases reported outside this period will be excluded.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Participants With MPS II	No Intervention	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants with Comorbidities Associated with MPS II	From January 1, 2008 to September 30, 2020	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).
Number of Participants Diagnosed With MPS II	From January 1, 2008 to September 30, 2020	Number of participants diagnosed with MPS II will be reported based on the retrospective data of participants collected from the database (DATASUS).
Age of MPS II Diagnosis	From January 1, 2008 to September 30, 2020	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).

Secondary Outcome Measures

Name	Time	Method
Number of Health Care Professionals Consulted	From January 1, 2008 to September 30, 2020	Number of health care professionals consulted before MPS II diagnosis will be reported based on the retrospective data of participants collected from the database (DATASUS).
Number of Health Care Professionals Who Diagnosed MPS II	From January 1, 2008 to September 30, 2020	Number of health care professionals who diagnosed the MPS II will be reported based on the retrospective data of participants collected from the database (DATASUS).
Number of Treatments Received Before and After MPS II Diagnosis	From January 1, 2008 to September 30, 2020	Number of treatments received by participant before and after MPS II diagnosis will be reported based on the retrospective data of participants collected from the database (DATASUS).
Time to MPS II Diagnoses	From January 1, 2008 to September 30, 2020	Time between first Unified Health System (a Brazilian Health System) consultation and MPS II diagnosis will be reported based on the retrospective data of participants collected from the database (DATASUS).
Time Between MPS Diagnosis II and Start of Treatment	From January 1, 2008 to September 30, 2020	Time between MPS diagnosis II and start of treatment will be reported based on the retrospective data of participants collected from the database (DATASUS).

Trial Locations

Locations (1)

Takeda Distribuidora Ltda; 🇧🇷 São Paulo, Brazil