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NCT Number: NCT05155488

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

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.

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Key information

Conditions

Mucopolysaccharidosis (MPS) Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Takeda Distribuidora Ltda

São Paulo, 04794-000, Brazil

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Exclusion criteria

MPS II cases reported outside this period will be excluded.

Treatment and study plan

No intervention

Other

This is a non-interventional study.

Primary outcomes

  1. Number of Participants Diagnosed With MPS II

    Time frame: 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).

  2. Number of Participants with Comorbidities Associated with MPS II

    Time frame: 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).

  3. Age of MPS II Diagnosis

    Time frame: 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 outcomes

  1. Number of Health Care Professionals Who Diagnosed MPS II

    Time frame: 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).

  2. Number of Health Care Professionals Consulted

    Time frame: 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).

  3. Time to MPS II Diagnoses

    Time frame: 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).

  4. Time Between MPS Diagnosis II and Start of Treatment

    Time frame: 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).

  5. Number of Treatments Received Before and After MPS II Diagnosis

    Time frame: 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).

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study title

Mucopolysaccharidosis Type II in Brazil: A Retrospective Secondary Database Study (Informatics Department of Brazilian Health System - DATASUS)

Important dates

Study start
2021
Primary completion
2022
Study completion
2022
First posted
Dec 13, 2021
Registry last updated
May 9, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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