Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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REBEC |
Last refreshed on:
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29 May 2023 |
Main ID: |
RBR-3rx9fj |
Date of registration:
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06/07/2015 |
Prospective Registration:
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No |
Primary sponsor: |
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Public title:
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A Study with additional chart review to evaluate clinical and biochemical characteristics and disease progression in patients with mucopolysaccharidosis type IIIB
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Scientific title:
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A prospective cross-sectional and longitudinal study with additional restrospective chart review to evaluate clinical and biochemical characteristics and disease progression in patients with mucopolysaccharidosis type IIIB
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Date of first enrolment:
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15/07/2014 |
Target sample size:
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Recruitment status: |
Recruitment completed |
URL:
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http://ensaiosclinicos.gov.br/rg/RBR-3rx9fj |
Study type:
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Observational |
Study design:
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Retrospective and prospective observational descritive study
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Phase:
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N/A
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Countries of recruitment
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Brazil
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Italy
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Portugal
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Spain
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United Kingdom
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United States
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Contacts
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Name:
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Anna Carolina
Coimbra |
Address:
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Rua da passagem, 123 / 6º andar, Botafogo
22290-030
Rio de Janeiro
Brazil |
Telephone:
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+55 (21) 3553 9763 |
Email:
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annacarolina.coimbra@incresearch.com |
Affiliation:
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INC Research BR Serviços de Pesquisas Clínicas Ltda. |
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Name:
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Anna Carolina
Coimbra |
Address:
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Rua da passagem, 123 / 6º andar, Botafogo
22290-030
Rio de Janeiro
Brazil |
Telephone:
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+55 (21) 3553 9763 |
Email:
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annacarolina.coimbra@incresearch.com |
Affiliation:
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INC Research BR Serviços de Pesquisas Clínicas Ltda. |
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Key inclusion & exclusion criteria
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Inclusion criteria: Definitive diagnosis of MPS IIIB; at least 1 years of age (biological age) at the time of written informed consent; understands the full nature and purpose of the study, including possible risks of study procedures; informed consent signature.
Exclusion criteria: Visual or hearing impairments sufficient to preclude cooperation with neurodevelopmental testing; history of poorly-controlled seizure disorder; currently receiving medication which, in the Investigator’s opinion, would be likely to substantially confound interpretation of the results, or has been on the current dose of psychotropic medication for less than 3 months; receiving a newly increased dose of melatonin (e.g., less than 3 months on current dose); previously received an investigational therapy for the treatment of MPS IIIB or has had hematopoietic stem cell transplant (HSCT); in the opinion of the Investigator, the patient has any other prior or ongoing medical condition that may present a safety risk, interfere with study compliance, or confound data interpretation.
Age minimum:
1Y
Age maximum:
Gender:
-
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Health Condition(s) or Problem(s) studied
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Mucopolysaccharidosis type IIIB
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E76.2
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Intervention(s)
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Other
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SP4.046.447.673
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This study will be conducted in patients with mucopolysaccharidosis type IIIB (MPS IIIB), and will comprise two components. The total number of subjects will be 43 and in Brazil it will be 15 patients. 1. Component 1 will involve an evaluation of the clinical characteristics of MPS IIIB in up to 30 patients based on a chart review and clinical assessments performed over a period of up to 6 weeks to facilitate scheduling. 2. Component 2 will involve an evaluation of the course of disease progression in a subset of approximately 15 patients who, after completing Component 1, will be followed for a period of at least 1 year and up to 3 years total. The routine clinical assessments will include neurological and developmental testing, blood and urine collection to laboratory assessments and medical and physical exams.
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E01.370.600
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E01.370.376.550
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E01.370.600.875
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SP4.006.057.488.719
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Primary Outcome(s)
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Descriptive comparison between patients who do/do not continue participation in the study into Component 2 made by summary of demographic data, selected medical history information, and baseline clinical and biochemical characteristics for all patients, after Component 1 of the study conclusion; Description of clinical characteristics, for the subset of patients participating in Component 2, made by summary of the observed values and changes from baseline over time, after study conclusion
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Secondary Outcome(s)
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Secundary outcomes are not expected
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Source(s) of Monetary Support
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Hospital de Clínicas de Porto Alegre
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INC Research BR Serviços de Pesquisas Clínicas Ltda.
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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