Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)NCT00214773
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
An observational study of Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome), run by BioMarin Pharmaceutical at 50 sites in 13 countries.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
- How many people
- 237 participants
- Where
- 50 sitesin 13 countries
- Study dates
- Jul 2005to 1 May 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
None listed.
Trial locations and contacts
50 sites in 13 countries.
50 sites in 13 countries
United States23 sites
Fullerton Genetic Center
Asheville, North Carolina, 28803
Johns Hopkins Univeristy School of Medicine
Baltimore, Maryland, 21287
Baton Rouge Clinic
Baton Rouge, Louisiana, 70808
Carolinas Medical Center
Charlotte, North Carolina, 28203
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Emory University
Decatur, Georgia, 30033
Indiana University School of Medicine
Indianapolis, Indiana, 46202
University of California, Irvine
Irvine, California, 92697
Children's Mercy Hospital
Kansas City, Missouri, 64108
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Children's Health Care
Minneapolis, Minnesota, 55404
University of Minnesota - Fairview University Medical Center
Minneapolis, Minnesota, 55455
Tulane University Medical Center
New Orleans, Louisiana, 70112
New York University
New York, New York, 10016
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
Children's Hospital and Research Center Oakland
Oakland, California, 94609
St. Joseph's Healthcare
Paterson, New Jersey, 07503
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health and Science University
Portland, Oregon, 97239
University of Utah Medical Center
Salt Lake City, Utah, 84132
Seattle Children's Hospital
Seattle, Washington, 98105
Sioux Valey Children's Speciality Clinics
Sioux Falls, South Dakota, 57117
Australia5 sites
Women and Children's Hospital
Adelaide, South Australia, 5006
Royal Children's Hospital, Brisbane
Brisbane, Queensland, 4029
Royal Children's Hospital
Parkville, Victoria
Princess Margaret Hospital for Children
Subiaco, Western Australia, 6008
Westmead Hospital
Wentworthville, New South Wales, NSW2145
Germany5 sites
Charité-Universitätsmedizin Berlin
Berlin, 13353
Universitätsklinikum Hamburg Eppendorf
Hamburg, 20246
Oberarzt Pädiatrie
Hanover, Lower Saxony, 30173
Universitatsklinikum Freiburg, Klinik II
Kammin, Mecklenburg-Vorpommern, 79106
Universitätsmedizin
Mainz, Rheinland-Pfalz RP, 55131
United Kingdom5 sites
Queen Elizabeth Hospital, University Hospitals Birmingham NHS
Birmingham, B29 6JD
Birmingham Children's Hospital, Steelhouse
Birmingham, B4 6NH
Great Ormond Street Hospital For Children, NHS Foundation Trust
London, WC1N 3JH
St Mary's Hospital, Willink Biochemical Genetics Unit
Manchester, M13 9WL
Salford Royal Hospital NHS Trust
Salford, M6 8HD
Italy3 sites
Azienda Ospedaliero Universitaria
Cibali, Catania, 95123
Università Milano Bicocca, Resp. Centro "Fondazione Mariani"
Monza, 20052
Department of Woman's and Child's Health
Padova, Veneto, 35128
France2 sites
Hôpital Femme Mère Enfant
Bron, Auvergne-Rhône-Alpes, 69677
Hopital Necker - Enfants Malades
Paris
Belgium1 site
Institute of Pathology and Genetics, Metabolic Unit
Charleroi, Wallonia, 6041
Lithuania1 site
Vilnius University Hospital, Santariskiu Klinikos
Vilnius, LT-08661
Netherlands1 site
Rotterdam University Hospital - Sophia's Children's Hospital
Rotterdam, 3015 GE
Portugal1 site
Hospital de Sao Joao, Unidae de Doencas Metabolicas
Porto, 4200-319
About this study
Official title: 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.
- Medical subject headings
- Mucopolysaccharidosis VI
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Observational | No intervention. This is an observational program. |
What is being measured
The primary outcome is the main question the study is designed to answer.
To further characterize the natural progression of MPS VI disease, irrespective of treatment modality and to evaluate efficacy and safety treatment with Galsulfase.
Measured: at least 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2005 (estimated)
- Study startJul 2005
- Primary completion1 May 2020
- Study completion1 May 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)Studies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 237 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Julie Johnson, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00214773
- Sponsor's protocol ID
- MPSVI CSP
- Status last verified
- Jan 2019
- First submitted
- 13 Sep 2005
- Last updated
- 5 Apr 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
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.
PubMed 27339555
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MPS VI Clinical Surveillance Program (CSP)
- ClinicalTrials.gov ID
- NCT00214773
- Acronym / sponsor ID
- MPSVI CSP
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 237 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Jul 2005
- Primary completion
- 1 May 2020 (actual)
- Study completion
- 1 May 2020 (actual)
- First posted
- 22 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observational | – | –No intervention. This is an observational program. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Apr 2021.
- Derived from this study: PubMed 27339555Solanki 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.
- BioMarin Pharmaceutical Inc WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Julie Johnson study director
BioMarin Pharmaceutical
50 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome), run by BioMarin Pharmaceutical at 50 sites in 13 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 50 sites in United States, Australia, Germany, United Kingdom, Italy and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00214773, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Apr 2021.