A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03227042|
Recruitment Status : Active, not recruiting
First Posted : July 24, 2017
Last Update Posted : January 28, 2021
|Condition or disease|
|Mucopolysaccharidosis Type IIIB|
|Study Type :||Observational|
|Actual Enrollment :||44 participants|
|Official Title:||A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)|
|Actual Study Start Date :||November 16, 2017|
|Estimated Primary Completion Date :||October 2024|
|Estimated Study Completion Date :||December 2024|
- Neurocognitive function [ Time Frame: Baseline + every 24 weeks for up to 192 weeks ]A neurodevelopmental assessment will be performed using standardized developmental tests to provide quantifiable measures of neurocognitive function.
- Behavioral function [ Time Frame: Baseline + every 24 weeks for up to 192 weeks ]Disease-related behaviors will be assessed using an MPS IIIB specific behavior rating scale.
- Quality of Life Tests [ Time Frame: Baseline + every 24 weeks for up to 192 weeks ]Multiple QoL tools will be used to capture physical, mental, and social well-being of the patient as well as to examine the impact of the patient's disease on the parent/guardian and family.
- Sleep habits [ Time Frame: Baseline + every 24 weeks for up to 192 weeks ]Patient sleep habits will be assessed using Children's Sleep Habits Questionnaires (CSHQ).
- Disease-specific Biomarkers [ Time Frame: Baseline + every 24 weeks for up to 192 weeks ]Urine sample for glycosaminoglycans (GAGs) and creatinine.
- Biochemical, Molecular, Cellular and Genetic Markers of Disease Burden [ Time Frame: Once (at baseline visit) ]Blood and urine samples will be used to evaluate biochemical, molecular cellular, and genetic/genomic aspects of MPS IIIB.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03227042
|United States, California|
|UCSF Benioff Children's Hospital Oakland|
|Oakland, California, United States, 94609|
|Hospital Universitario Austral|
|Pilar, Argentina, B1629AHJ|
|Murdoch Childrens Research Institute and Royal Children's Hospital|
|Melbourne, Victoria, Australia, 3052|
|Medical Genetics Service/HCPA, Department of Genetics/UFRGS|
|Pôrto Alegre, Rio Grande Do Sul, Brazil, 90035-903|
|Fundación Cardio Infantil - Instituto de Cardiología|
|University Medical Center Hamburg-Eppendorf|
|Hamburg, Germany, 20246|
|Hospital Clínico Universitario de Santiago|
|Santiago de Compostela, A Coruña, Spain, 15706|
|MacKay Memorial Children's Hospital|
|Taipei, Taiwan, 10449|
|Gazi University Faculty of Medicine|
|Ankara, Turkey, 06560|
|Study Director:||Medical Director, MD||Allievex Medical Monitor|