Mucopolysaccharidosis type IIIB
Conditions
Interventions
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
Other
SP4.006.057.488.719
Sponsors
Hospital de Clínicas de Porto Alegre
INC Research BR Serviços de Pesquisas Clínicas Ltda.
Eligibility
Age
1 Years to No maximum
Inclusion criteria
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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secundary outcomes are not expected | — |
Countries
Brazil, Italy, Portugal, Spain, United Kingdom, United States
Contacts
Public ContactAnna Coimbra
INC Research BR Serviços de Pesquisas Clínicas Ltda.
Outcome results
None listed