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)

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.

Study Overview

• Status: Completed
• Conditions: Mucopolysaccharidosis (MPS)
• Intervention / Treatment: Other: No Intervention

• Study Type: Observational
• Enrollment (Actual): 250

Contacts and Locations

Study Locations

• Brazil
  • São Paulo, Brazil, 04794-000
    • Takeda Distribuidora Ltda

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Participants diagnosed with MPS II cases in Brazil.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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.	Other: No Intervention; This is a non-interventional study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Health Care Professionals Who Diagnosed MPS II	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).	From January 1, 2008 to September 30, 2020
Number of Health Care Professionals Consulted	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).	From January 1, 2008 to September 30, 2020
Time to MPS II Diagnoses	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).	From January 1, 2008 to September 30, 2020
Time Between MPS Diagnosis II and Start of Treatment	Time between MPS diagnosis II and start of treatment 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 Treatments Received Before and After MPS II Diagnosis	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).	From January 1, 2008 to September 30, 2020

Collaborators and Investigators

• Sponsor: Takeda

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): December 6, 2021
• Primary Completion (Actual): February 15, 2022
• Study Completion (Actual): February 15, 2022

Study Registration Dates

• First Submitted: November 30, 2021
• First Submitted That Met QC Criteria: November 30, 2021
• First Posted (Actual): December 13, 2021

Study Record Updates

• Last Update Posted (Actual): May 9, 2022
• Last Update Submitted That Met QC Criteria: May 6, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidosis II; Mucopolysaccharidoses
• Other Study ID Numbers: TAK-765-5001; MACS-2021-021601 (Other Identifier: Takeda)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.
• IPD Sharing 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.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No