MPS IIIA
Surrogate Endpoint Trial (SET) for Individuals with MPS IIIA
The recent protocol amendment now allows for patients greater than one year of age to be eligible for participation.
SET is a one-year, multi-center study designed to study the natural progression of MPS IIIA, in approximately 20 patients.
Over a period of twelve months, participants in the study will be evaluated to assess the severity and progression of MPS IIIA, as measured by developmental age and milestones, central nervous system (CNS) function (including cognition, speech and motor skills) and biochemical markers of the condition (levels of heparan sulfate and its breakdown products in blood urine and cerebrospinal fluid (CSF).
Additional information can be found on www.clinicaltrials.gov (identifier NCT01047306), or contact:
| Shire HGT Medical Information | or | Amy K. Fisher, MS, CGC |
| Tel: (484) 595-8850 | Shire HGT Global Medical Affairs | |
| HGTmedcomm@shire.com | Tel: (857) 413-9553 | |
| afisher@shire.com |
MPS IIIA & MPS IIIB
This research project will focus on how the immune system is affected in patients with MPS IIIA or MPS IIIB. A blood test will be done to measure blood cell count. To participate, patients must:
Be less than or exactly 20 years of age with a confirmed diagnosis of MPS IIIA or MPS IIIB
Not use medications that suppress the immune system at time of enrollment
Not have respiratory, urinary or other infections at the time of enrollment
The study will take place at your local healthcare providers office or at The Research Institute at Nationwide Childrens Hospital, Columbus, OH. For more information and to make an appointment, contact Chelsea Rankin at 614-355-2897 or Chelsea.rankin@nationwidechildrens.org .
MPS IV
MorCAP (MPS IVA, Morquio A Clinical Assessment Program)
MorCAP [Study MOR-001] has been designed to provide a better clinical understanding of the natural history of MPS IVA (Morquio A) syndrome by measuring various aspects of the disorder including endurance and respiratory function in affected patients. These insights may help BioMarin Pharmaceutical Inc. design future clinical studies. Participation in the MorCAP study will require one or more visits yearly to a clinic or hospital for up to 10 years. Experimental drug will not be administered during these visits. MorCAP is a multinational study and includes sites located in the United States, United Kingdom, South America, Canada, Europe and Asia. For further information, please call toll-free 866-961-8212 and a BioMarin representative will respond to your questions regarding MorCAP.
