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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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
        • Hopital Necker - Enfants Malades
    • Rhône-alpes
      • Bron cedex, Rhône-alpes, France, 69677
        • Hopital Femme Mere Enfant
  • Germany
      • Berlin, Germany, 13353
        • Charité-Universitätsmedizin 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Observational
No intervention. This is an observational program.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
BioMarin Pharmaceutical

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 1, 2005

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 1, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 1, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 13, 2005

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 14, 2005

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 22, 2005

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 5, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 2, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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