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Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

This study is currently recruiting participants.
Verified April 2017 by BioMarin Pharmaceutical
Sponsor:
ClinicalTrials.gov Identifier:
NCT00214773
First Posted: September 22, 2005
Last Update Posted: April 21, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
BioMarin Pharmaceutical
  Purpose
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.

Condition
Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: MPS VI Clinical Surveillance Program (CSP)

Resource links provided by NLM:


Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • 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 ]

Biospecimen Retention:   Samples Without DNA
Urine and serum

Estimated Enrollment: 200
Study Start Date: July 2005
Estimated Study Completion Date: December 2020
Estimated Primary Completion Date: July 2020 (Final data collection date for primary outcome measure)
Groups/Cohorts
Observational
No intervention. This is an observational program.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00214773


Contacts
Contact: MEDINFO MEDINFO (615) 523-0310 MEDINFO@bmrn.com

  Hide Study Locations
Locations
United States, California
University of California, Irvine Recruiting
Irvine, California, United States, 92697
Contact: Margaret Knight    949-824-0521    mnrobins@uci.edu   
Principal Investigator: Virginia Kimonis, MD         
Children's Hospital of Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Ellynore Florendo    323-361-2178    eflorendo@chla.usc.edu   
Principal Investigator: Linda Randolph, MD         
Children's Hospital and Research Center Oakland Recruiting
Oakland, California, United States, 94609
Contact: Jo Ann Johnson    510-428-3885 ext 5421    jajohnson@mail.cho.org   
Principal Investigator: Paul Harmatz, MD         
United States, Georgia
Emory University Recruiting
Decatur, Georgia, United States, 30033
Contact: Alison Akard    404-778-8562    alison.b.akard@emory.edu   
Principal Investigator: William Wilcox, MD         
United States, Illinois
Ann and Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Katherine Kim    312-227-6765    kkim@luriechildrens.org   
Principal Investigator: Barbara Burton, MD         
United States, Indiana
Indiana University School of Medicine Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Susan Romie    317-278-6650    sromie@iu.edu   
Principal Investigator: Bryan Hainline, MD         
United States, Louisiana
Baton Rouge Clinic Recruiting
Baton Rouge, Louisiana, United States, 70808
Contact: Cheri Wittmer    225-246-9913    cwittmer@brclinic.com   
Principal Investigator: Kevin Babin, MD         
Tulane University Medical Center Terminated
New Orleans, Louisiana, United States, 70112
United States, Maryland
Johns Hopkins Univeristy School of Medicine Recruiting
Baltimore, Maryland, United States, 21287
Contact: Ada Hamosh    410-614-7021      
Principal Investigator: Ada Hamosh, MD         
United States, Minnesota
Children's Health Care Recruiting
Minneapolis, Minnesota, United States, 55404
Contact: Brianna McMichael    612-813-6291    Brianna.McMichael@childrensmn.org   
Principal Investigator: Gunther Scharer, MD         
University of Minnesota - Fairview University Medical Center Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Brenda Diethelm-Okita    612-625-1594    dieth001@umn.edu   
Principal Investigator: Chester Whitley, MD         
United States, Missouri
Children's Mercy Hospital Recruiting
Kansas City, Missouri, United States, 64108
Contact: Kristen Williams    816-760-5591    knwilliams@cmh.edu   
Principal Investigator: Bryce Heese, MD         
United States, New Jersey
St. Joseph's Healthcare Recruiting
Paterson, New Jersey, United States, 07503
Contact: JoAnne Daguanno    973-754-3072    daguannj@sjhmc.org   
Principal Investigator: Punita Gupta, MD         
United States, New York
New York University Recruiting
New York, New York, United States, 10016
Contact: Marissa Ferraris    212-263-0139    swapnil.parmar@nyumc.org   
Principal Investigator: Heather Lau, MD         
United States, North Carolina
Fullerton Genetic Center Recruiting
Asheville, North Carolina, United States, 28803
Contact: Kerry Crandall    828-213-0027    Kerry.Crandall@msj.org   
Principal Investigator: Chad Haldeman-Englert, MD         
Carolinas Medical Center Recruiting
Charlotte, North Carolina, United States, 28203
Contact: Sara Sagui    704-381-6819    sara.sagui@carolinashealthcare.org   
Principal Investigator: Edward Spence, MD         
United States, Oregon
Oregon Health and Science University Terminated
Portland, Oregon, United States, 97239
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Leigh Ann Higa    215-590-3692    higal@email.chop.edu   
Principal Investigator: Can Ficicioglu, MD         
Sub-Investigator: Paige Kaplan, MD         
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Jennifer Baker    412-692-6378    jennifer.baker@chp.edu   
Principal Investigator: Gerard Vockley, MD         
United States, South Dakota
Sioux Valey Children's Speciality Clinics Recruiting
Sioux Falls, South Dakota, United States, 57117
Contact: Alycia Brantz    605-312-0061    Alycia.brantz@sanfordhealth.org   
Principal Investigator: Laura Davis-Keppen, MD         
United States, Utah
University of Utah Medical Center Recruiting
Salt Lake City, Utah, United States, 84132
Contact: Carrie Bailey    801-587-3605    carrie.bailey@hsc.utah.edu   
Principal Investigator: Nicola Longo, MD         
United States, Virginia
Children's Hospital of the King's Daughters Recruiting
Norfolk, Virginia, United States, 23507
Contact: Angela Casey    757-668-9818    angela.casey@chkd.org   
Principal Investigator: Samantha Vergano, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Linnea Brody    206-884-1264    Linnea.brody@seattlechildrens.org   
Principal Investigator: Angela Sun, MD         
Australia, New South Wales
Westmead Hospital Terminated
Wentworthville, New South Wales, Australia, NSW2145
Australia, Queensland
Royal Children's Hospital, Brisbane Terminated
Brisbane, Queensland, Australia, 4029
Australia, South Australia
Women and Children's Hospital Terminated
Adelaide, South Australia, Australia, 5006
Australia, Victoria
Royal Children's Hospital Terminated
Parkville, Victoria, Australia
Australia, Western Australia
Princess Margaret Hospital for Children Terminated
Subiaco, Western Australia, Australia, 6008
Austria
LKH-Universitätsklinik Graz, Kinderklinik Recruiting
Graz, Steiermark, Austria, A-8036
Contact: Michaela Brunner-Krainz, MD    +43-316-385-2813    michaela.brunner@klinikum-graz.at   
Principal Investigator: Michaela Brunner-Krainz, MD         
Belgium
Institute of Pathology and Genetics, Metabolic Unit Recruiting
Charleroi, Wallonie, Belgium, 6041
Contact: Dominique Roland, MD    +3271.47.30.47    dominique.roland@ipg.be   
Principal Investigator: Dominique Roland, MD         
France
Hôpital Femme Mère Enfant Recruiting
Bron cedex, Rhône-alpes, France, 69677
Contact: Nathalie Guffon, MD    +33 472129537    nathalie.guffon-fouilhoux@chu-lyon.fr   
Principal Investigator: Nathalie Guffon, MD         
Hopital Necker - Enfants Malades Recruiting
Paris, France
Contact: Anais Brassier    +00-331-444-94731    anais.brassier@nck.aphp.fr   
Principal Investigator: Anais Brassier, MD         
Germany
Universitatsklinikum Freiburg, Klinik II Recruiting
Kammin, Mecklenburg-vorpommern, Germany, 79106
Contact: Ursula Wein    0049(0)761/270-44740    ursula.wein@uniklinik-freiburg.de   
Principal Investigator: Rudolf Korinthenberg, MD         
Oberarzt Pädiatrie Recruiting
Hannover, Niedersachsen, Germany, 30173
Contact: Kay Müller-Deile, MD    +49 511 8115-33 23    mueller-deile@hka.de   
Principal Investigator: Kay Müller-Deile, MD         
Universitätsmedizin Recruiting
Mainz, Rheinland-Pfalz RP, Germany, 55131
Contact: Maria Ann Winter    +49 (0) 6131 17-4579    mawinter@unimedizin-mainz.de   
Principal Investigator: Julia Hennermann, MD         
Charité-Universitätsmedizin Berlin Recruiting
Berlin, Germany, 13353
Contact: Nikolaus Tiling    0049-(0)30-450-553824    nikolaus.tiling@charite.de   
Principal Investigator: Ursula Plockinger, MD         
Universitätsklinikum Hamburg Eppendorf Recruiting
Hamburg, Germany, 20246
Contact: Maria Lippold    +49-40-7410-53710    m.lippold@uke.di   
Principal Investigator: Nicole Muschol, MD         
Ireland
MidWestern Regional Hospital Recruiting
Limerick, Ireland
Contact: Ina Knerr, MD       ina.knerr@cuh.ie   
Principal Investigator: Ina Knerr, MD         
Italy
Azienda Ospedaliero Universitaria Recruiting
Cibali, Catania, Italy, 95123
Contact: Rita Barone    +39.347.682.5365    rbarone@iol.it   
Principal Investigator: Rita Barone, MD         
Department of Woman's and Child's Health Recruiting
Padova, Veneto, Italy, 35128
Contact: Nicolletta Gasparotto    +39-049-8218070    nicoletta.gasparotto@gmail.com   
Principal Investigator: Giorgio Perilongo, MD         
Università Milano Bicocca, Resp. Centro "Fondazione Mariani" Recruiting
Monza, Italy, 20052
Contact: Stefania Sala    +39.039.2334915    stefania.sala@hsgerardo.org   
Principal Investigator: Rossella Parini, MD         
Lithuania
Vilnius University Hospital, Santariskiu Klinikos Recruiting
Vilnius, Lithuania, LT-08661
Contact: Loretta Cimbalistiene, MD    +370-5-2501792    Loretta.Cimbalistiene@santa.lt   
Principal Investigator: Loretta Cimbalistiene, MD         
Netherlands
Rotterdam University Hospital - Sophia's Children's Hospital Recruiting
Rotterdam, Netherlands, 3015 GE
Contact: Audrey Vollebregt    31107032076    a.vollebregt@erasmusmc.nl   
Principal Investigator: Ans van der Ploeg, MD         
Portugal
Hospital de Sao Joao, Unidae de Doencas Metabolicas Recruiting
Porto, Portugal, 4200-319
Contact: Elisa Leao Teles, MD    35.122.5089.138    e.leaoteles@hsjoao.min-saude.pt   
Principal Investigator: Elisa Leao Teles, MD         
Sweden
Astrid Lindgrens Children's Hospital Recruiting
Stockholm, Sweden
Contact: Ylva Beckman    +46.8.5858.7417    Ylva.beckman@karolinska.se'   
Principal Investigator: Karin Naess, MD         
United Kingdom
Queen Elizabeth Hospital, University Hospitals Birmingham NHS Recruiting
Birmingham, United Kingdom, B29 6JD
Contact: Katherine Peers    +07918023336    Katherine.Peers@uhb.nhs.uk   
Principal Investigator: Tarekegn G Hiwot, MD         
Birmingham Children's Hospital, Steelhouse Recruiting
Birmingham, United Kingdom, B4 6NH
Contact: Alice Stewart    0121.333.9954    Alice.Stewart@bch.nhs.uk   
Principal Investigator: Saikat Santra, MD         
Great Ormond Street Hospital For Children, NHS Foundation Trust Recruiting
London, United Kingdom, WC1N 3JH
Contact: Misbah Alhadad    +020-7829-7867 ext 7867    Misbah.Alhadad@gosh.nhs.uk   
Principal Investigator: Maureen Cleary, MD         
St Mary's Hospital, Willink Biochemical Genetics Unit Recruiting
Manchester, United Kingdom, M13 9WL
Contact: Jane Roberts    +44161-701-2300    Jane.roberts2@cmft.nhs.uk   
Principal Investigator: Jane Roberts, MD         
Salford Royal Hospital NHS Trust Recruiting
Salford, United Kingdom, M6 8HD
Contact: Marie Meehan    0161.206.4192    marie.meehan@srft.nhs.uk   
Principal Investigator: Reena Sharma, MD         
Sponsors and Collaborators
BioMarin Pharmaceutical
  More Information

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT00214773     History of Changes
Other Study ID Numbers: MPSVI CSP
First Submitted: September 13, 2005
First Posted: September 22, 2005
Last Update Posted: April 21, 2017
Last Verified: April 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis VI
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases