Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)



Status:Recruiting
Conditions:Endocrine, Metabolic
Therapuetic Areas:Endocrinology, Pharmacology / Toxicology
Healthy:No
Age Range:Any
Updated:1/23/2019
Start Date:July 2005
End Date:December 2020
Contact:MEDINFO MEDINFO
Email:MEDINFO@bmrn.com
Phone:(615) 523-0310

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Trial Summary
Trial Overview
Inclusion Criteria

MPS VI Clinical Surveillance Program (CSP)

The objectives of this program are: to further characterize the natural progression of MPS VI
disease; to generate and disseminate information on the care and management of MPS VI
patients to clinical and medical professionals; to provide a resource to physicians and
patients by providing information for optimizing patient care based on aggregate data; to
characterize the clinical response to long-term Naglazyme® (galsulfase) treatment; to further
characterize the long-term safety of Naglazyme® treatment.


Inclusion Criteria

All patients must meet the following criteria to qualify for enrollment in the CSP:

- Patient or patient's parent or legal guardian, if child is under 18 year old or is
unable to consent, has provided a signed Patient Information and Authorization Form.

- Patient has laboratory results confirming a diagnosis of MPS VI disease based on
detection of deficient ARSB activity (on fibroblasts, leucocytes or dried blood
spots)and/or abnormality on the ARSB gene.

- Patient is willing to undergo general assessments to establish baseline data or
permits physician to enter assessment data recorded prior to CSP entry if available in
the patient's medical records. General assessments include: urinary GAG level, urinary
protein level, serum sample for antibody levels, height, weight, and patient history.
We found this trial at
24
sites
Children's Hospital of Pittsburgh
3414 Fifth Avenue
Pittsburgh, Pennsylvania 15213
Principal Investigator: Gerard Vockley, MD
Phone: 412-692-6378
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Carolinas Medical Center
1000 Blythe Blvd
Charlotte, North Carolina 28203
(704) 355-2000
Principal Investigator: Edward Spence, MD
Phone: 704-381-6819
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Charlotte, NC
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Children's Mercy Hospital
2401 Gillham Rd
Kansas City, Missouri 64108
(816) 234-3000
Principal Investigator: Bryce Heese, MD
Phone: 816-760-5591
Children's Mercy Hospital Children's Mercy Hospitals and Clinics continues redefining pediatric medicine throughout the Midwest...
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Childrens Hospital Los Angeles
4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Principal Investigator: Linda Randolph, MD
Phone: 323-361-2178
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Children's Hospital of The King's Daughters
601 Children's Lane
Norfolk, Virginia 23507
(757) 668-7000
Principal Investigator: Samantha Vergano, MD
Phone: 757-668-9818
Children's Hospital of The King's Daughters Children
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Children's Hospital and Research Center Oakland
747 52nd St
Oakland, California 94609
(510) 428-3000
Principal Investigator: Paul Harmatz, MD
Phone: 510-428-3885
Children's Hospital and Research Center Oakland For nearly 100 years, Children's Hospital & Research Center...
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Oakland, CA
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Children's Hospital of Philadelphia
South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Can Ficicioglu, MD
Phone: 215-590-3692
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Philadelphia, PA
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Oregon Health and Science University
3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Principal Investigator: David Koeller, MD
Phone: 503-418-1061
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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Portland, OR
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Fullerton Genetic Center
Asheville, North Carolina 28803
Principal Investigator: Chad Haldeman-Englert, MD
Phone: 828-213-0027
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Asheville, NC
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Johns Hopkins Univeristy School of Medicine
Baltimore, Maryland
Principal Investigator: Ada Hamosh, MD
Phone: 410-614-7021
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Baltimore, MD
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Baton Rouge Clinic
Baton Rouge, Louisiana 70808
Principal Investigator: Kevin Babin, MD
Phone: 225-246-9913
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Baton Rouge, LA
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Ann & Robert H. Lurie Children's Hospital of Chicago
225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Barbara Burton, MD
Phone: 312-227-6765
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Chicago, IL
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Emory University
Decatur, Georgia 30033
Principal Investigator: William Wilcox, MD
Phone: 404-778-8562
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Decatur, GA
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Indiana University School of Medicine
340 W 10th St #6200
Indianapolis, Indiana 46202
(317) 274-3772
Principal Investigator: Bryan Hainline, MD
Phone: 317-278-6650
Indiana University School of Medicine With more than 2,000 students in 2013, the Indiana University...
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Indianapolis, IN
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University of California, Irvine
Irvine, California 92697
949-824-5011
Principal Investigator: Virginia Kimonis, MD
Phone: 949-824-0521
University of California, Irvine Since 1965, the University of California, Irvine has combined the strengths...
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Irvine, CA
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Children's Health Care
Minneapolis, Minnesota 55404
Principal Investigator: Gunther Scharer, MD
Phone: 612-813-6291
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Minneapolis, MN
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University of Minnesota Fairview University Medical Center
Minneapolis, Minnesota 55455
Principal Investigator: Chester Whitley, MD
Phone: 612-625-1594
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Minneapolis, MN
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Tulane University Medical Center
New Orleans, Louisiana 70112
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New Orleans, LA
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New York University
70 Washington Square S
New York, New York 10012
(212) 998-1212
Principal Investigator: Heather Lau, MD
Phone: 212-263-0139
New York University More than 175 years ago, Albert Gallatin, the distinguished statesman who served...
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St. Joseph's Healthcare
Paterson, New Jersey 07503
Principal Investigator: Punita Gupta, MD
Phone: 973-754-3072
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Paterson, NJ
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University of Utah Medical Center
Salt Lake City, Utah 84132
Principal Investigator: Nicola Longo, MD
Phone: 801-587-3605
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Salt Lake City, UT
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Seattle Children's Hospital
4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Principal Investigator: Angela Sun, MD
Phone: 206-884-1264
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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Sioux Valey children's Speciality Clinics
Sioux Falls, South Dakota 57104
Principal Investigator: Laura Davis-Keppen, MD
Phone: 605-312-0061
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Sioux Falls, SD
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Westmead Hospital
Wentworthville, New South Wales 2145
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