A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
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|ClinicalTrials.gov Identifier: NCT03227042|
Recruitment Status : Recruiting
First Posted : July 24, 2017
Last Update Posted : May 9, 2018
|Condition or disease|
|Mucopolysaccharidosis Type IIIB|
|Study Type :||Observational|
|Estimated Enrollment :||60 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 :||July 31, 2022|
|Estimated Study Completion Date :||November 15, 2022|
- 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
|Contact: BioMarin Trial Inquiriesfirstname.lastname@example.org|
|United States, California|
|UCSF Benioff Children's Hospital Oakland||Recruiting|
|Oakland, California, United States, 94609|
|Contact: Christopher Luc 510-428-3885 ext 4785 email@example.com|
|Principal Investigator: Paul Harmatz, MD|
|Murdoch Childrens Research Institute and Royal Children's Hospital||Recruiting|
|Melbourne, Victoria, Australia, 3052|
|Contact: Shannon Kokoszka +61 3 9936 6157 firstname.lastname@example.org|
|Principal Investigator: Heidi Peters, MD|
|Medical Genetics Service/HCPA, Department of Genetics/UFRGS||Recruiting|
|Pôrto Alegre, Rio Grande Do Sul, Brazil, 90035-903|
|Contact: Larissa Da Silva +55 51 3359 6366 email@example.com|
|Principal Investigator: Roberto Giugliani, MD|
|University Medical Center Hamburg-Eppendorf||Recruiting|
|Hamburg, Germany, 20246|
|Contact: Martina Lippold 49-40-7410-53710 firstname.lastname@example.org|
|Contact: Julia Voelker 00 email@example.com|
|Principal Investigator: Nicole Muschol, MD|
|Hospital Clínico Universitario de Santiago||Recruiting|
|Santiago de Compostela, A Coruña, Spain, 15706|
|Contact: María José De Castro +34.622.214.171.124 firstname.lastname@example.org|
|Principal Investigator: Maria Luz Couce, MD|
|MacKay Memorial Children's Hospital||Recruiting|
|Taipei, Taiwan, 10449|
|Contact: Youhsin Huang +886-2-25433535 ext 3089 email@example.com|
|Principal Investigator: Shuan-Pei Lin, MD|
|Gazi University Faculty of Medicine||Recruiting|
|Ankara, Turkey, 06560|
|Contact: Mine Tutkal +90 505 253 86 34 firstname.lastname@example.org|
|Principal Investigator: Ilyas Okur, MD|
|Study Director:||Medical Director, MD||BioMarin Medical Monitor|