Mucopolysaccharidosis (MPS)NCT05155488
A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil
An observational study of Mucopolysaccharidosis (MPS), run by Takeda at 1 site in Brazil.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis (MPS)
- How many people
- 250 participants
- Where
- 1 siteBrazil
- Study dates
- 6 Dec 2021to 15 Feb 2022
- 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: Participants diagnosed with MPS II cases in Brazil.
Who may be able to join
- 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.
Who cannot join
- MPS II cases reported outside this period will be excluded.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
- Conditions
- Mucopolysaccharidosis (MPS)
- Being tested
- No Intervention
- Medical subject headings
- Mucopolysaccharidoses
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 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. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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).
Measured: 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).
Measured: From January 1, 2008 to September 30, 2020
Secondary outcomes (5)
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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: From January 1, 2008 to September 30, 2020
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2021
- Study start6 Dec 2021
- Primary completion15 Feb 2022
- Study completion15 Feb 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis (MPS)Studies 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
- Case only
- Time perspective
- Retrospective
- Enrolment
- 250 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
- NCT05155488
- Sponsor's protocol ID
- TAK-765-5001
- Other IDs
- MACS-2021-021601
- Status last verified
- May 2022
- First submitted
- 30 Nov 2021
- Last updated
- 9 May 2022
- 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 9 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mucopolysaccharidosis Type II in Brazil: A Retrospective Secondary Database Study (Informatics Department of Brazilian Health System - DATASUS)
- ClinicalTrials.gov ID
- NCT05155488
- Acronym / sponsor ID
- TAK-765-5001
- Conditions
- Mucopolysaccharidosis (MPS)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 250 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 6 Dec 2021 (actual)
- Primary completion
- 15 Feb 2022 (actual)
- Study completion
- 15 Feb 2022 (actual)
- First posted
- 13 Dec 2021 (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.
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 design
- Observational model
- Case only
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants With MPS II | – | Other: No InterventionRetrospective 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants diagnosed with MPS II cases in Brazil.
Key inclusion criteria (1 listed)
- 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.
Key exclusion criteria (1 listed)
- MPS II cases reported outside this period will be excluded.
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 MPS II |
Primary outcome
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).
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).
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).
5 secondary outcomes
Number of Health Care Professionals Who Diagnosed MPS II
Time frame: From January 1, 2008 to September 30, 2020
Number of Health Care Professionals Consulted
Time frame: From January 1, 2008 to September 30, 2020
Time to MPS II Diagnoses
Time frame: From January 1, 2008 to September 30, 2020
Time Between MPS Diagnosis II and Start of Treatment
Time frame: From January 1, 2008 to September 30, 2020
Number of Treatments Received Before and After MPS II Diagnosis
Time frame: From January 1, 2008 to September 30, 2020
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2022.
- To obtain more information on the study, click here/on this linkLink 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.
Related trials for Mucopolysaccharidosis (MPS)
Active, not recruitingObservational study
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
RecruitingPhase 1/2
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
Mucopolysaccharidosis Type IIIA
Enrolling by invitationPhase 2/3
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
Mucopolysaccharidosis II
RecruitingObservational study
Cardiac Structure and Function in MPS
MPS I, MPS IV A
Not yet recruitingObservational study
MPS IIIA Disease Monitoring Program
MPS IIIA, Sanfilippo Syndrome
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 Mucopolysaccharidosis (MPS), run by Takeda at 1 site in Brazil.
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 1 inclusion and 1 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: finished, as of the sponsor's last update on 9 May 2022. 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 Brazil. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of Participants Diagnosed With MPS II (time frame: From January 1, 2008 to September 30, 2020). The study lists 3 primary outcomes in all. 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 9 May 2022.