A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
| Status: | Recruiting |
|---|---|
| Conditions: | Other Indications, Metabolic |
| Therapuetic Areas: | Pharmacology / Toxicology, Other |
| Healthy: | No |
| Age Range: | Any - 18 |
| Updated: | 8/31/2018 |
| Start Date: | November 16, 2017 |
| End Date: | November 15, 2022 |
| Contact: | BioMarin Trial Inquiries |
| Email: | medinfo@bmrn.com |
| Phone: | 1-800-983-4587 |
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 BioMarin in
this study.
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 BioMarin in
this study.
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
We found this trial at
2
sites
Oakland, California 94609
Principal Investigator: Paul Harmatz, MD
Phone: 510-428-3885
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50 Flemington Road
Melbourne, Victoria 3052
Melbourne, Victoria 3052
Principal Investigator: Heidi Peters, MD
Phone: +61 3 9936 6157
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