Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

April 2, 2021 updated by: BioMarin Pharmaceutical

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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

237

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Wentworthville, New South Wales, Australia, NSW2145
        • Westmead Hospital
    • Queensland
      • Brisbane, Queensland, Australia, 4029
        • Royal Children's Hospital, Brisbane
    • South Australia
      • Adelaide, South Australia, Australia, 5006
        • Women and Children's Hospital
    • Victoria
      • Parkville, Victoria, Australia
        • Royal Children's Hospital
    • Western Australia
      • Subiaco, Western Australia, Australia, 6008
        • Princess Margaret Hospital for Children
  • Austria
    • Steiermark
      • Graz, Steiermark, Austria, A-8036
        • LKH-Universitätsklinik Graz, Kinderklinik
  • Belgium
    • Wallonie
      • Charleroi, Wallonie, Belgium, 6041
        • Institute of Pathology and Genetics, Metabolic Unit
  • France
      • Paris, France
        • Hôpital Necker - Enfants Malades
    • Rhône-alpes
      • Bron cedex, Rhône-alpes, France, 69677
        • Hôpital Femme Mère Enfant
  • Germany
      • Berlin, Germany, 13353
        • Charite-Universitatsmedizin Berlin
      • Hamburg, Germany, 20246
        • Universitatsklinikum Hamburg Eppendorf
    • Mecklenburg-vorpommern
      • Kammin, Mecklenburg-vorpommern, Germany, 79106
        • Universitatsklinikum Freiburg, Klinik II
    • Niedersachsen
      • Hannover, Niedersachsen, Germany, 30173
        • Oberarzt Pädiatrie
    • Rheinland-Pfalz RP
      • Mainz, Rheinland-Pfalz RP, Germany, 55131
        • Universitätsmedizin
  • Ireland
      • Limerick, Ireland
        • Midwestern Regional Hospital
  • Italy
      • Monza, Italy, 20052
        • Università Milano Bicocca, Resp. Centro "Fondazione Mariani"
    • Catania
      • Cibali, Catania, Italy, 95123
        • Azienda Ospedaliero Universitaria
    • Veneto
      • Padova, Veneto, Italy, 35128
        • Department of Woman's and Child's Health
  • Lithuania
      • Vilnius, Lithuania, LT-08661
        • Vilnius University Hospital, Santariskiu Klinikos
  • Netherlands
      • Rotterdam, Netherlands, 3015 GE
        • Rotterdam University Hospital - Sophia's Children's Hospital
  • Portugal
      • Porto, Portugal, 4200-319
        • Hospital de Sao Joao, Unidae de Doencas Metabolicas
  • Sweden
      • Stockholm, Sweden
        • Astrid Lindgrens Children's Hospital
  • United Kingdom
      • Birmingham, United Kingdom, B29 6JD
        • Queen Elizabeth Hospital, University Hospitals Birmingham NHS
      • Birmingham, United Kingdom, B4 6NH
        • Birmingham Children's Hospital, Steelhouse
      • London, United Kingdom, WC1N 3JH
        • Great Ormond Street Hospital For Children, NHS Foundation Trust
      • Manchester, United Kingdom, M13 9WL
        • St Mary's Hospital, Willink Biochemical Genetics Unit
      • Salford, United Kingdom, M6 8HD
        • Salford Royal Hospital NHS Trust
  • United States
    • California
      • Irvine, California, United States, 92697
        • University of California, Irvine
      • Los Angeles, California, United States, 90027
        • Children's Hospital of Los Angeles
      • Oakland, California, United States, 94609
        • Children's Hospital and Research Center Oakland
    • Georgia
      • Decatur, Georgia, United States, 30033
        • Emory University
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann and Robert H. Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University School of Medicine
    • Louisiana
      • Baton Rouge, Louisiana, United States, 70808
        • Baton Rouge Clinic
      • New Orleans, Louisiana, United States, 70112
        • Tulane University Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins Univeristy School of Medicine
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Children's Health Care
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota - Fairview University Medical Center
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Children's Mercy Hospital
    • New Jersey
      • Paterson, New Jersey, United States, 07503
        • St. Joseph's Healthcare
    • New York
      • New York, New York, United States, 10016
        • New York University
    • North Carolina
      • Asheville, North Carolina, United States, 28803
        • Fullerton Genetic Center
      • Charlotte, North Carolina, United States, 28203
        • Carolinas Medical Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh
    • South Dakota
      • Sioux Falls, South Dakota, United States, 57117
        • Sioux Valey Children's Speciality Clinics
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah Medical Center
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Children's Hospital of The King's Daughters
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Observational
No intervention. This is an observational program.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To further characterize the natural progression of MPS VI disease, irrespective of treatment modality and to evaluate efficacy and safety treatment with Galsulfase.
Time Frame: at least 15 years
at least 15 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioMarin Pharmaceutical

Investigators

  • Study Director: Julie Johnson, BioMarin Pharmaceutical

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2005

Primary Completion (Actual)

May 1, 2020

Study Completion (Actual)

May 1, 2020

Study Registration Dates

First Submitted

September 13, 2005

First Submitted That Met QC Criteria

September 14, 2005

First Posted (Estimate)

September 22, 2005

Study Record Updates

Last Update Posted (Actual)

April 5, 2021

Last Update Submitted That Met QC Criteria

April 2, 2021

Last Verified

January 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MPSVI CSP

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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