MPS VI Clinical Surveillance Program (CSP)

Status: Completed
Location: See all (50) locations...
Study Type: Observational
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: No
View:

• 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.

Locations
United States
California
University of California, Irvine
Irvine
Children's Hospital of Los Angeles
Los Angeles
Children's Hospital and Research Center Oakland
Oakland
Georgia
Emory University
Decatur
Illinois
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago
Indiana
Indiana University School of Medicine
Indianapolis
Louisiana
Baton Rouge Clinic
Baton Rouge
Tulane University Medical Center
New Orleans
Maryland
Johns Hopkins Univeristy School of Medicine
Baltimore
Minnesota
Children's Health Care
Minneapolis
University of Minnesota - Fairview University Medical Center
Minneapolis
Missouri
Children's Mercy Hospital
Kansas City
North Carolina
Fullerton Genetic Center
Asheville
Carolinas Medical Center
Charlotte
New Jersey
St. Joseph's Healthcare
Paterson
New York
New York University
New York
Oregon
Oregon Health and Science University
Portland
Pennsylvania
Children's Hospital of Philadelphia
Philadelphia
Children's Hospital of Pittsburgh
Pittsburgh
South Dakota
Sioux Valey Children's Speciality Clinics
Sioux Falls
Utah
University of Utah Medical Center
Salt Lake City
Virginia
Children's Hospital of the King's Daughters
Norfolk
Washington
Seattle Children's Hospital
Seattle
Other Locations
Australia
Women and Children's Hospital
Adelaide
Royal Children's Hospital, Brisbane
Brisbane
Royal Children's Hospital
Parkville
Princess Margaret Hospital for Children
Subiaco
Westmead Hospital
Wentworthville
Austria
LKH-Universitätsklinik Graz, Kinderklinik
Graz
Belgium
Institute of Pathology and Genetics, Metabolic Unit
Charleroi
France
Hôpital Femme Mère Enfant
Bron Cedex
Hopital Necker - Enfants Malades
Paris
Germany
Charité-Universitätsmedizin Berlin
Berlin
Universitätsklinikum Hamburg Eppendorf
Hamburg
Oberarzt Pädiatrie
Hannover
Universitatsklinikum Freiburg, Klinik II
Kammin
Universitätsmedizin
Mainz
Ireland
MidWestern Regional Hospital
Limerick
Italy
Azienda Ospedaliero Universitaria
Cibali
Università Milano Bicocca, Resp. Centro Fondazione Mariani
Monza
Department of Woman's and Child's Health
Padova
Lithuania
Vilnius University Hospital, Santariskiu Klinikos
Vilnius
Netherlands
Rotterdam University Hospital - Sophia's Children's Hospital
Rotterdam
Portugal
Hospital de Sao Joao, Unidae de Doencas Metabolicas
Porto
Sweden
Astrid Lindgrens Children's Hospital
Stockholm
United Kingdom
Birmingham Children's Hospital, Steelhouse
Birmingham
Queen Elizabeth Hospital, University Hospitals Birmingham NHS
Birmingham
Great Ormond Street Hospital For Children, NHS Foundation Trust
London
St Mary's Hospital, Willink Biochemical Genetics Unit
Manchester
Salford Royal Hospital NHS Trust
Salford
Time Frame
Start Date: July 2005
Completion Date: May 1, 2020
Participants
Target number of participants: 237
Treatments
Observational
No intervention. This is an observational program.
Sponsors
Leads: BioMarin Pharmaceutical

This content was sourced from clinicaltrials.gov