Clinical trial NCT00214773: Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
Official title: MPS VI Clinical Surveillance Program (CSP)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2005-09-22
Enrollment: 237
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients with a confirmed diagnosis of MPS VI disease may participate in the CSP. It is not a requirement that the patients enrolled in the CSP receive Galsulfase 1mg/kg to participate as this is an observational program.

Brief 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 criteria: 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.

Primary outcomes:
measure: To further characterize the natural progression of MPS VI disease, irrespective of treatment modality and to evaluate efficacy and safety treatment with Galsulfase.
timeFrame: at least 15 years
Secondary outcomes:
Other outcomes:
Locations:
city: Irvine
country: United States
facility: University of California, Irvine
state: California
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: Oakland
country: United States
facility: Children's Hospital and Research Center Oakland
state: California
city: Decatur
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University School of Medicine
state: Indiana
city: Baton Rouge
country: United States
facility: Baton Rouge Clinic
state: Louisiana
city: New Orleans
country: United States
facility: Tulane University Medical Center
state: Louisiana
city: Baltimore
country: United States
facility: Johns Hopkins Univeristy School of Medicine
state: Maryland
city: Minneapolis
country: United States
facility: Children's Health Care
state: Minnesota
city: Minneapolis
country: United States
facility: University of Minnesota - Fairview University Medical Center
state: Minnesota
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Paterson
country: United States
facility: St. Joseph's Healthcare
state: New Jersey
city: New York
country: United States
facility: New York University
state: New York
city: Asheville
country: United States
facility: Fullerton Genetic Center
state: North Carolina
city: Charlotte
country: United States
facility: Carolinas Medical Center
state: North Carolina
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh
state: Pennsylvania
city: Sioux Falls
country: United States
facility: Sioux Valey Children's Speciality Clinics
state: South Dakota
city: Salt Lake City
country: United States
facility: University of Utah Medical Center
state: Utah
city: Norfolk
country: United States
facility: Children's Hospital of the King's Daughters
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Wentworthville
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Brisbane
country: Australia
facility: Royal Children's Hospital, Brisbane
state: Queensland
city: Adelaide
country: Australia
facility: Women and Children's Hospital
state: South Australia
city: Parkville
country: Australia
facility: Royal Children's Hospital
state: Victoria
city: Subiaco
country: Australia
facility: Princess Margaret Hospital for Children
state: Western Australia
city: Graz
country: Austria
facility: LKH-Universitätsklinik Graz, Kinderklinik
state: Steiermark
city: Charleroi
country: Belgium
facility: Institute of Pathology and Genetics, Metabolic Unit
state: Wallonie
city: Bron cedex
country: France
facility: Hôpital Femme Mère Enfant
state: Rhône-alpes
city: Paris
country: France
facility: Hopital Necker - Enfants Malades
city: Kammin
country: Germany
facility: Universitatsklinikum Freiburg, Klinik II
state: Mecklenburg-vorpommern
city: Hannover
country: Germany
facility: Oberarzt Pädiatrie
state: Niedersachsen
city: Mainz
country: Germany
facility: Universitätsmedizin
state: Rheinland-Pfalz RP
city: Berlin
country: Germany
facility: Charité-Universitätsmedizin Berlin
city: Hamburg
country: Germany
facility: Universitätsklinikum Hamburg Eppendorf
city: Limerick
country: Ireland
facility: MidWestern Regional Hospital
city: Cibali
country: Italy
facility: Azienda Ospedaliero Universitaria
state: Catania
city: Padova
country: Italy
facility: Department of Woman's and Child's Health
state: Veneto
city: Monza
country: Italy
facility: Università Milano Bicocca, Resp. Centro "Fondazione Mariani"
city: Vilnius
country: Lithuania
facility: Vilnius University Hospital, Santariskiu Klinikos
city: Rotterdam
country: Netherlands
facility: Rotterdam University Hospital - Sophia's Children's Hospital
city: Porto
country: Portugal
facility: Hospital de Sao Joao, Unidae de Doencas Metabolicas
city: Stockholm
country: Sweden
facility: Astrid Lindgrens Children's Hospital
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital, University Hospitals Birmingham NHS
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital, Steelhouse
city: London
country: United Kingdom
facility: Great Ormond Street Hospital For Children, NHS Foundation Trust
city: Manchester
country: United Kingdom
facility: St Mary's Hospital, Willink Biochemical Genetics Unit
city: Salford
country: United Kingdom
facility: Salford Royal Hospital NHS Trust
Study officials:
affiliation: BioMarin Pharmaceutical
name: Julie Johnson
role: STUDY_DIRECTOR
References:
citation: Solanki GA, Sun PP, Martin KW, Hendriksz CJ, Lampe C, Guffon N, Hung A, Sisic Z, Shediac R, Harmatz PR; CSP Study Group. Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP). Mol Genet Metab. 2016 Aug;118(4):310-8. doi: 10.1016/j.ymgme.2016.06.001. Epub 2016 Jun 3.