A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

DesconocidoClinicalTrials.gov

ID: NCT03227042Tipo: OBSERVATIONALInicio: 16 de nov de 2017Fin estimado: 1 de dic de 2025

Traducción no disponible, mostrando original

Resumen

This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B). Mucopolysaccharidosis type IIIB is a severe neurodegenerative disorder. The information gathered from this trial may help inform the design and interpretation of subsequent interventional studies. No clinical intervention or study drug is provided by Allievex in this study.

Elegibilidad

Edad máxima: 18 YearsSexo: ALL

Criterios de inclusión

Have deficient NAGLU enzyme activity at Baseline. Blood for NAGLU enzyme activity will be collected and analyzed centrally.
Is up to 18 years of age
Written informed consent from parent or legal guardian and assent from subject, if required
Has the ability to comply with protocol requirements, in the opinion of the investigator

Criterios de exclusión

Has another neurological illness that may have caused cognitive decline (e.g., trauma, meningitis, or hemorrhage) before study entry
Has received stem cell, gene therapy, or enzyme replacement therapy for MPS IIIB
Has received any investigational medication within 30 days prior to the Baseline visit or is scheduled to receive any investigational drug during the course of the study
Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with protocol requirements, the subject's wellbeing or safety, or the interpretability of the subject's clinical data.
Is currently participating in another natural history study

Ubicaciones

Hospital Universitario Austral

Pilar, Argentina

Patrocinadores

PrincipalAllievex Corporation

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