A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Completed

Conditions studied: Mucopolysaccharidosis Type IIIB, Mucopolysaccharidosis Type 3 B, MPS III B, MPS 3 B

In brief

Mucopolysaccharidosis type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B) is a severe neurodegenerative disorder. The purpose of this study is to learn more about the health problems in patients with MPS IIIB and how to measure these problems over time. It will particularly look at how the disease develops in young children. This is an observational study, so no experimental drug will be given. The results from this study will help us design future studies to measure whether these health problems get better when we give experimental drug for MPS IIIB.

Key facts

Study ID

NCT02493998

Run by

Allievex Corporation

People needed

22

Starts

2015-11-01

Expected to finish

2019-04-01

Last updated by the study team

2020-03-06

Who can join

Age: 1 and older, up to 10. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Individuals eligible to participate in this study must meet all of the following criteria:
• Has deficient NAGLU enzyme activity at Screening. Blood for NAGLU enzyme activity will be collected and analyzed centrally.
• Is ≥ 1 and ≤ 10 years of age and has an age-equivalent of ≥ 12 months on the VABS-II
• DQ ≥ 50 (determined by BSID-III or KABC-II)
• Has presented with signs/symptoms consistent with MPS IIIB; for individuals who have not presented with signs/symptoms of disease (e.g., siblings of known patients), the determination of eligibility will be at the discretion of the BioMarin medical monitor in conjunction with the site investigator.
• 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

You may not qualify if…

• Has another neurological illness that may have caused cognitive decline (e.g., trauma, meningitis, or hemorrhage) before study entry
• Requires ventilation support, except for noninvasive support at night
• Has received stem cell, gene therapy or ERT for MPS IIIB
• Has contraindications for neurosurgery (e.g., congenital heart disease, severe respiratory impairment, or clotting abnormalities)
• Has contraindications for MRI scans (e.g., cardiac pacemaker, metal fragment or chip in the eye, or aneurysm clip in the brain)
• Has a history of poorly controlled seizure disorder
• Is prone to complications from intraventricular drug administration, including patients with hydrocephalus or ventricular shunts
• 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.

Where it is running

• Children's Hospital and Research Center Oakland — Oakland, California, United States
• Melbourne Children's Trials Centre — Melbourne, Victoria, Australia
• Fundacion Cardioinfantil-Instituto de Cardiologia — Bogotá, Colombia
• University Medical Center Hamburg Eppendorf, Department of Pediatrics — Hamburg, Germany
• Hospital Clinico Universitario de Santiago — Santiago de Compostela, Spain
• MacKay Memorial Children's Hospital — Taipei, Taiwan
• Gazi University Faculty of Medicine — Ankara, Turkey (Türkiye)
• Somers Clinical Research Facility, Great Ormond Street Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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