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A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) (MARS)

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ClinicalTrials.gov Identifier: NCT02294877
Recruitment Status : Recruiting
First Posted : November 19, 2014
Last Update Posted : August 17, 2018
Sponsor:
Collaborator:
ICON plc
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Brief Summary:
The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural history of MPS IVA; to evaluate the long-term effectiveness and safety of Vimizim®, including, but not limited to, the occurrence of serious hypersensitivity reactions, anaphylaxis, and changes in antibody status; to help the medical community with the development of recommendations for monitoring MPS IVA patients and reports on patient outcomes to optimize patient care; to collect data on other treatment paradigms, and evaluate the prevalences of their use and their effectiveness; to characterize the effects and safety of Vimizim treatment 5 years from enrollment in the Registry for patients under 5 years of age; to monitor pregnancy exposure, including maternal, neonatal, and infant outcomes; and to monitor patients who have completed the MOR-005 and MOR-007 clinical trials. These patients will be encouraged to enroll in the applicable Registry Substudy and will be monitored using the MOR-005 and MOR-007 assessment schedules, respectively.

Condition or disease Intervention/treatment
Mucopolysaccharidosis IV Type A Morquio A Syndrome MPS IVA Drug: Vimizim® (elosulfase alfa)

Detailed Description:

MARS is a multicenter, multinational, observational disease registry for patients diagnosed with Mucopolysaccharidosis Type IVA (MPS IVA). The Registry will collect medical history, and clinical and safety assessments every six months, for up to 10 years. The Registry will enroll and collect data on patients over a period of at least 8 years from the time of the first marketing approval globally and data on individual patients will continue to be collected for at least 2 years from the time the last patient was enrolled or until the Registry is completed.

These assessments are designed to further characterize the spectrum of clinical signs and symptoms of the disease, and to further characterize the safety profile of Vimizim in a broader population. It is not required that patients receive Vimizim to be eligible to participate in this Registry.

In addition, this Registry will collect additional data on patients who have completed the MOR-005 and MOR-007 clinical trials. The MOR-005 and MOR-007 clinical trial patients will be enrolled into the appropriate Registry Substudy for a minimum of 5 years from the time of the patient's enrollment in the MOR-005 clinical study or MOR-007 clinical study. After the 5 year period, these patients should remain in MARS until the Registry is complete.


Study Type : Observational [Patient Registry]
Estimated Enrollment : 583 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 10 Years
Official Title: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Study Start Date : September 2014
Estimated Primary Completion Date : September 2024
Estimated Study Completion Date : September 2024



Intervention Details:
  • Drug: Vimizim® (elosulfase alfa)
    Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS)
    Other Name: BMN 110


Primary Outcome Measures :
  1. Safety of Vimizim treated patients as measured by the incidences of Adverse Events, Serious Adverse Events, and immunology tests [ Time Frame: 10 Years ]

    Additional safety measures are medications, occurrence of Bone Marrow Transplant/Hematopoietic Stem Cell Transplant, clinical laboratory tests, vital signs, Electrocardiograms, Echocardiograms, immunogenicity results, physical examinations, imaging studies, and cervical spine imaging.

    All assessments in this observational study will be carried out per the participating institution's standard of care.

    The MARS Annual report will descriptively summarize the safety and outcome measurements for the duration of this voluntary, observational study.


  2. Efficacy of Vimizim as measured by the 6MWT, 3MSCT, RFTs, FEV1, FIVC, FVC, and MVV [ Time Frame: 10 years ]
    Efficacy of Vimizim as measured by the changes in the 6 minute walk test (6MWT), 3 minute stir climb test (3MSCT), respiratory function tests (RFTs) including forced expiratory volume for 1 second (FEV1), forced inspiratory vital capacity (FIVC), forced vital capacity (FVC), and maximum voluntary ventilation (MVV) and by urinary Keratan Sulfate.


Secondary Outcome Measures :
  1. The MARS Pregnancy Substudy will collect safety data measured by Adverse Events, Serious Adverse Events, immunology tests, and the infant outcomes as measured by a live birth [ Time Frame: 10 years ]
    The MARS Pregnancy Substudy will collect safety data on the outcomes of pregnancy in women who receive Vimizim as measured by Adverse Events, Serious Adverse Events, immunology tests, and the infant outcomes as measured by a live birth.


Biospecimen Retention:   Samples Without DNA
Blood serum; urine samples


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, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with MPS IVA disease and patients treated with Vimizim, although patients are not required to receive Vimizim to be eligible to participate in this Registry.
Criteria

Inclusion Criteria:

Patients eligible to participate in this Registry must meet all of the following criteria:

  • Diagnosed with MPS IVA as confirmed by either N-acetylgalactosamine 6-sulfatase (GALNS) enzymatic test or by a diagnostic molecular test
  • Willing and able to provide written, signed informed consent, or, in the case of patients age < 18 years, provide written assent (if required) and written informed consent, signed by a legally authorized representative after the nature of the Registry has been explained and prior to performance of any Registry-related procedures
  • Willing to undergo assessments to establish baseline data or permit Investigator to enter assessment data recorded prior to Registry entry if available in the patient's medical records. Entry assessments may include: demographics, medical history, urinary keratan sulfate level, urinary protein level, immunogenicity testing, vital signs, physical examination, and height and weight

Patients eligible to participate in the Registry Substudy for MOR-005 must meet all of the following criteria:

  • Must have completed the MOR-005 clinical trial
  • Willing and able to provide written, signed informed consent, or, in the case of patients age < 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the Registry Substudy has been explained and prior to any Registry-related Substudy procedures
  • Willing to permit Investigator to enter assessment data recorded prior to Registry
  • Substudy entry if available in the patient's medical records

Patients eligible to participate in this Registry Substudy for MOR-007 must meet all of the following criteria:

  • Must have completed the MOR-007 clinical trial
  • Willing and able to provide written, signed informed consent, or in the case of patients age < 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the Registry Substudy has been explained, and prior to any Registry-related Substudy procedures
  • Willing to permit Investigator to enter assessment data recorded prior to Registry Substudy entry if available in the patient's medical records

Exclusion Criteria:

Patients who meet the following exclusion criterion will not be eligible to participate in the Registry or Registry Substudies:

• Patients currently participating in a BMN 110 (elosulfase alfa) clinical trial


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): NCT02294877


Contacts
Contact: MEDINFO 615-523-0310 MEDINFO@bmrn.com
Contact: BioMarin 415-506-6700

  Hide Study Locations
Locations
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Contact: Grace Goode    501-364-3120    goodega@archildrens.org   
Principal Investigator: Thomas Burrow, MD         
Sub-Investigator: Elizabeth A Sellars, MD         
United States, California
University of Southern California Recruiting
Los Angeles, California, United States, 90033
Contact: Kathryn Moseley, MS, RD    323-226-3816    kmoseley@yahoo.com   
Contact: Maria Ramirez    323-409-6800    mramirez@dhs.lacounty.gov   
Principal Investigator: Shoji Yano, MD         
Children's Hospital of Orange County Recruiting
Orange, California, United States, 92868
Contact: Nina Movsesyan, Phd    714-509-3008    nmovsesyan@choc.org   
Principal Investigator: Raymond Wang, MD         
Sub-Investigator: Richard Chang, MD         
United States, Delaware
A.I. Dupont Hospital for Children Recruiting
Wilmington, Delaware, United States, 19803
Contact: Lauren Davey    302-651-5916    LDavey@nemours.org   
Principal Investigator: Michael Bober, MD         
Sub-Investigator: William Mackenzie, MD         
Sub-Investigator: Colleen Ditro, APN         
Sub-Investigator: Angela Duker         
United States, District of Columbia
Children's National Health System Recruiting
Washington, District of Columbia, United States, 20010
Contact: Katie Crosby, CGC    202-476-6812    kacrosby@childrensnational.org   
Principal Investigator: Pranoot Tanpaiboon, MD         
United States, Florida
Miami Children's Hospital Recruiting
Miami, Florida, United States, 33155
Contact: Michelin Janvier    786-624-3534    michelin.janvier@mch.com   
Contact: Claudia Rodriguez Paez    786-268-1741    claudia.rodriguezpaez@mch.com   
Principal Investigator: Mislen Bauer, MD         
United States, Georgia
Emory University Recruiting
Decatur, Georgia, United States, 30033
Contact: Stephanie Cagle    404-778-8618    scagle@emory.edu   
Contact: Elizabeth Sablon       esablon@emory.edu   
Principal Investigator: William Wilcox, MD         
United States, Hawaii
Kapiolani Medical Center for Women and Children Recruiting
Honolulu, Hawaii, United States, 96826
Contact: Micah Tong    808-983-6427    micah.tong@hawaiipacifichealth.org   
Principal Investigator: Robert Wallerstein, MD         
Sub-Investigator: Michah Tong, CCRP         
Sub-Investigator: JoAnn Cheung, MA         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital Recruiting
Chicago, Illinois, United States, 60611
Contact: Rachel Katz, MS    312-227-6764    rkatz@luriechildrens.org   
Principal Investigator: Barbara Burton, MD         
Sub-Investigator: Joel Charrow, MD         
Sub-Investigator: Lauren Hitchens, APN         
Sub-Investigator: Erika Vucko, APN         
Sub-Investigator: Shanna Widera, APN         
United States, Kentucky
University of Louisville Research Foundation Inc. - Kosair Charities Pediatric Clinical Research Unit Recruiting
Louisville, Kentucky, United States, 40202
Contact: Sarah Penny    502-629-2721    slpenn03@louisville.edu   
Contact: Molly Harper    502-629-7447    molly.harper@louisville.edu   
Principal Investigator: Alexander Asamoah, MD         
Sub-Investigator: Janice Sullivan, MD         
Sub-Investigator: Kara Goodin, MD         
United States, Massachusetts
Children's Hospital of Boston Recruiting
Boston, Massachusetts, United States, 02115
Contact: Kyla Almedia    857-218-4636    kyla.almeida@childrens.harvard.edu   
Contact: Abra Roberts    617-919-2536    abra.roberts@childrens.harvard.edu   
Principal Investigator: Amy Kritzer, MD         
Sub-Investigator: Olaf Bodamer, MD         
Sub-Investigator: Leah Hecht         
Sub-Investigator: Kyla Almeida         
Sub-Investigator: Abra Roberts         
Sub-Investigator: Vera Anastasoaie         
United States, Minnesota
Childrens Hospitals and Clinics of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55404
Contact: Alissa Jorgenson    612-813-6922    alissa.jorgenson@childrensmn.org   
Principal Investigator: Vikas Bhambani, MD         
Sub-Investigator: Nancy J. Mendelsohn, MD         
Sub-Investigator: Rebecca Olson         
United States, Nevada
Children's Specialty Center of Nevada Recruiting
Las Vegas, Nevada, United States, 89109
Contact: Daniel Crosier    702-862-1136    dcrosier@cure4thekids.org   
Principal Investigator: Angela Berg, DNP         
Sub-Investigator: Ayyanar Kanyalakshmi, MD         
Sub-Investigator: Arlene Bayreder, MSN         
Sub-Investigator: Alan Ikeda, MD         
Sub-Investigator: McKenzie Karelus, DPT         
Sub-Investigator: Nicola Longo, MD         
Sub-Investigator: Nik Farhana Nik Abdul Rashid, MD         
United States, New York
NYU-Langone Medical Center School of Medicine Recruiting
New York, New York, United States, 10016
Contact: Marissa Ferraris    212-263-0139    Marissa.Ferraris@nyumc.org   
Principal Investigator: Heather Lau, MD         
Sub-Investigator: Zahra Bakhtiar         
Sub-Investigator: Marissa Ferraris, RN         
Sub-Investigator: Danika Anganoo         
Sub-Investigator: Michele Ford         
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
Contact: Crista Walters    919-681-1945    crista.walters@duke.edu   
Principal Investigator: Priya Kishnani, MD         
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Genevieve Nesom    267-426-1368    nesomg@email.chop.edu   
Principal Investigator: Lynne Ierardi-Curto, MD         
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Nadene Henderson    412-692-6065    nadene.henderson@chp.edu   
Principal Investigator: Damara Ortiz, MD         
Sub-Investigator: Areeg El-Gharbawy, MD         
Sub-Investigator: Gerard Vockley, MD         
Sub-Investigator: Nadene Henderson, MS, LCGC         
United States, Virginia
Children's Hospital of the King's Daughters Recruiting
Norfolk, Virginia, United States, 23507
Contact: Corina Moorehead    757-401-1132    acmoorehead@gmail.com   
Contact: Carrie E Mishoe    304-942-2234      
Principal Investigator: Samantha A Vergano, MD         
Sub-Investigator: Erin McAndrews, LPN         
Sub-Investigator: Jennifer Parrott, LPN         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98145-5005
Contact: Linnea Brody    206-884-1264    linnea.brody@seattlechildrens.org   
Principal Investigator: Angela Sun, MD         
Sub-Investigator: Klane White, MD         
Sub-Investigator: Susan Hale, MN, ARNP         
United States, Wisconsin
Children's Hospital of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Cassie Bird    414-266-3072    cbird@mcw.edu   
Principal Investigator: Donald Basel, MD         
Sub-Investigator: Stephanie Offord         
Sub-Investigator: William Rhead, MD         
Australia, New South Wales
Children's Hospital of Westmead Recruiting
Westmead, New South Wales, Australia, 2145.0
Contact: Kaustuv Bhattacharya, MD    +61 (2) 9845 3650    kaustuvb@chw.edu.au   
Principal Investigator: Kaustuv Bharracharya, MD         
Australia, Queensland
Chidren's Heatlh Queensland Hospital and Health Service Recruiting
South Brisbane, Queensland, Australia, 4101.0
Contact: Anita Inwood    +61 (0)7 3068 4426    Anita.Inwood@health.qld.gov.au   
Principal Investigator: Anita Inwood, NP         
Australia, South Australia
Royal Adelaide Hospital Recruiting
Adelaide, South Australia, Australia, 5000.0
Contact: Janice Fletcher, MD    +61 8 8161 8742    Janice.Fletcher@health.sa.gov.au   
Principal Investigator: Janice Fletcher, MD         
Australia, Victoria
Royal Children's Hospital - Victoria Recruiting
Parkville, Victoria, Australia, 3052.0
Contact: Heidi Peters, MD    61 3 8341 6200    heidi.peters@mcri.edu.au   
Principal Investigator: Heidi Peters, MD         
Austria
Medical University of Vienna Recruiting
Vienna, Austria, 1090
Contact: Charlotte de Gier       charlotte.degier@meduniwien.ac.at   
Principal Investigator: Vassiliki Konstantopoulo, MD         
Sub-Investigator: Dorothea Moeslinger, MD         
Belgium
Antwerp University Hospital Recruiting
Edegem, Belgium, 2650
Contact: Tine Maes    0032(0)318214155    tine.maes@uaz.be   
Principal Investigator: Francois Eyskens, MD         
Canada, British Columbia
Children's & Women's Centre of British Columbia Recruiting
Vancouver, British Columbia, Canada, V52 4H4
Contact: Anita Thomas    604-875-3806    athomas2@cw.bc.ca   
Principal Investigator: Lorne Clarke, MD         
Sub-Investigator: Sylvia Stockler-Ispiroglu, MD         
Sub-Investigator: Sandra Sirrs, MD         
Canada, Quebec
Sainte-Justine Hospital Recruiting
Montreal, Quebec, Canada, H3T 1C5
Contact: Fabiola Breault    514-345-4931 ext 2774    fabiola.breault@reserche-ste-justine.qc.ca   
Principal Investigator: Phillipe Campeau, MD         
McGill University Health Centre Recruiting
Montreal, Quebec, Canada, H4A 3J1
Contact: Sandra Pepin    514-9341934 ext 23737    sandra.pepin@muhc.mcgill.ca   
Contact: Hasmig Kavoukian    514-934-1934 ext 35661    hasmig.kavoukian@muhc.mcgill.ca   
Principal Investigator: John Mitchell, MD         
Université de Sherbrooke Recruiting
Sherbrooke, Quebec, Canada, J1H 5N4
Contact: Francide Lapointe    819-346-1110 ext 12802    flapointe.chus@ssss.gouv.qc.ca   
Principal Investigator: Bruno Maranda, MD         
Sub-Investigator: Sebastien Levesque, MD         
Czechia
Klinika Dětského a Dorostového Lékařstvi Recruiting
Prague, Czechia
Contact: Jiri Zeman    22496 7777      
Principal Investigator: Martin Magner, MD         
Sub-Investigator: Jiri Zeman         
Denmark
Copenhagen University Hospital, Klinik For Sjaeldne Handicap Recruiting
Copenhagen, Dk-2100, Denmark
Contact: Sannie Mahncke    +45 23866095    sannie.mahncke@region.dk   
Principal Investigator: Merete Ljugberg, MD         
France
Hôpital Femme Mère Enfant Recruiting
Bron Cedex, France, 69677
Contact: Nicole Chatagnat    +33472129541    nicole.chatagnat@chu-lyon.fr   
Contact: Nathalie Reyes    +33 4 72 12 95 41      
Principal Investigator: Nathalie Guffon, MD         
Hospital d'enfants, Service de Medecine Infantile I Recruiting
Vandœuvre-lès-Nancy, France, 54500.0
Contact: Francois Feillet, MD    03 83 15 4796    f.feillet@chu-nancy.fr   
Principal Investigator: Francois Feillet, MD         
Germany
Clinical Study Center, University Pediatric Clinic Recruiting
Bochum, Germany, 4791
Contact: Sandra Boeger    0234-509-2809    s.boeger@klinikum-Bochum.de   
Principal Investigator: Thomas Lücke, MD         
Universitätsklinikum Hamburg-Eppendorf Recruiting
Hamburg, Germany, 20246
Contact: Nicole Muschol, MD    +49-40-7410-49321    muschol@uke.uni-hamburg.de   
Principal Investigator: Nicole Muschol, MD         
Zentrum für seltene Erkrankungen, Horst-Schmidt-Kliniken Recruiting
Wiesbaden, Germany, 65199
Contact: Maurizio Scarpa    0611-43-2314    Maurizio.scarpa@hsk-wiesbaden.de   
Principal Investigator: Christina Lampe, MD         
Ireland
Temple Street Children's University Hospital Recruiting
Dublin, Ireland
Contact: Eithne Losty    01 8921903    eithne.losty@cuh.ie   
Principal Investigator: Ardeshir Ahmad Monavari, MD         
Sub-Investigator: Ellen Crushell, MD         
Sub-Investigator: Ina Knerr, MD         
Sub-Investigator: Joanne Hughes, MD         
Italy
Azienda Policlinico Vittorio Emanuele, presidio G Rodolico, Università di Catania Recruiting
Catania, Italy, 95123
Contact: Agata Fiumara, MD    3900953782478      
Principal Investigator: Agata Fiumara, MD         
Sub-Investigator: Viviana Brafa, MD         
Sub-Investigator: Annarita Pittala, MD         
Sub-Investigator: Federica Raudino, MD         
Azienda Ospedaliero Meyer Recruiting
Firenze, Italy, 50132
Contact: Alice Donati, MD    0039 055 5662482    m.donati@meyer.it   
Principal Investigator: Alice Donati, MD         
Sub-Investigator: Elena Procopio, MD         
Azienda Ospedaliero San Gerardo di Monza Recruiting
Monza, Italy, 20090
Contact: Giusi Sgroi    390392334915 ext 3525    gsgroi@fondazionembbm.it   
Principal Investigator: Rossella Parini, MD         
Sub-Investigator: Cinzia Galimberti, MD         
Sub-Investigator: Serena Gasperini, MD         
Sub-Investigator: Emanuela Marrocco, MD         
Sub-Investigator: Serena Motto, MD         
Sub-Investigator: Miriam Rigoldo, MD         
Sub-Investigator: Stefania Sala, MD         
Federico II University Hospital Recruiting
Naples, Italy, 80131
Contact: Emma Coppola       emma.coppola@studenti.unina.it   
Principal Investigator: Roberto Della Casa, MD         
Sub-Investigator: Simona Fecarotta, MD         
Malaysia
Hospital Kuala Lumpur Recruiting
Kuala Lumpur, Malaysia, 50586
Contact: Siti Nuradila Bt Muhamad    +60-326155555 ext 7062    lutfan_aakif@yahoo.com   
Contact: Mohamed Ali Noraishah       dr_aishah@hotmail.com   
Principal Investigator: Ngu Hock Lock, MD         
Sub-Investigator: Hui Bein Chew         
Sub-Investigator: Huey Yin Leong         
Netherlands
Academic Medical Center Recruiting
Amsterdam, Netherlands, 1105 AZ
Contact: Frits Wijburg, MD    31 20 5662303    f.a.wijburg@amc.nl   
Principal Investigator: Frits Wijburg, MD         
Rotterdam University Hospital Medical Centre Recruiting
Rotterdam, Netherlands, 3015 GE
Contact: Jacqueline Hardon    +31 0 107037047    j.hardon@erasmusmc.nl   
Principal Investigator: Ana Van der Ploeg, MD         
Poland
The Children Memorial Health Institute Recruiting
Warszawa, Poland, 04-730
Contact: Anna Tylki-Szymanska, MD    48 22 815-7584    a.tylki@czd.pl   
Principal Investigator: Anna Tylki-Szymanska, MD         
Portugal
Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra) Recruiting
Coimbra, Portugal, 3000-062
Contact: Paula Garcia, MD    +351 239 239 488 700    pg@chuc.min-saude.pt   
Principal Investigator: Paula Garcia, MD         
Sub-Investigator: Luisa Diogo, MD         
Centro Hospitalar Lisboa Norte Recruiting
Lisboa, Portugal
Contact: Diogo Nuno Fonseca da Cruz, MD       diogocruz@yahoo.com   
Principal Investigator: Diogo Nuno Fonseca da Cruz, MD         
Sub-Investigator: Patricio Ricardo da Terra Aguiar         
Centro Hospitalar Lisboa Centro - Hospital de Dona Estefânia Recruiting
Lisbon, Portugal, 1169-045
Contact: Silvia Sequeira, MD    +351 21 312 68 94    silviasequeira@gmail.com   
Principal Investigator: Silvia Sequeira, MD         
Sub-Investigator: Ana Cristina Ferreira, MD         
Puerto Rico
San Jorge Children's Hospital, Centro de Investigaciones Clinicas Recruiting
San Juan, Puerto Rico, 00912
Contact: Heriberto Roman-Berrios    787-999-9453    heriberto.roman@sanjorgechildrenshospital.com   
Contact: Mirta Fernandez    787-999-9453    mirta.fernandez@sanjorgechildrenshospital.com   
Principal Investigator: Juan A Santiago-Cornier, MD         
Taiwan
MacKay Memorial Hospital Recruiting
Taipei, Taiwan, 10449
Contact: Shuan-Pei Lin, MD    886-2-2543-3535 ext 3089    4535lin@gmail.com   
Principal Investigator: Shuan-Pei Lin, MD         
United Kingdom
Belfast City Hospital Recruiting
Belfast, United Kingdom, BT9 7AB
Contact: Alison Murphy    02890636366    alison.murphy@belfasttrust.hscni.net   
Principal Investigator: Fiona Stewart, MD         
University Hospital Birmingham - Selly Oaks Recruiting
Birmingham, United Kingdom, B15 2WB
Contact: Shaun Bolton    44121-371-6795    shaun.bolton@uhb.nhs.uk   
Contact: Vishy Veeranna, MD    +44 0 121 371 6985    vishy.veeranna@uhb.nhs.uk   
Principal Investigator: Tarekegn G Hiwot, MD         
Sub-Investigator: Charlotte Dawson, MD         
Birmingham Children's Hospital Recruiting
Birmingham, United Kingdom, B4 6NH
Contact: Alice Stewart    0121 333 9907    Alice.stewart@bch.nhs.uk   
Contact: Alison Berry    +0121-333-9962    Alison.Berry@bch.nhs.uk   
Principal Investigator: Saikat Santra, MA/MB/BCh         
Sub-Investigator: Suresh Vijayaraghavan, MB         
Royal Free NHS Foundation Trust Recruiting
London, United Kingdom, NW3 2QG
Contact: Erika Lansdell    020-7794-0500 ext 33476    erika.lansdell@nihr.ac.uk   
Principal Investigator: Derralynn Hughes, MD         
Sub-Investigator: Uma Ramaswami, MD         
Sub-Investigator: Atulkumar Mehta, MD         
National Hospital for Neurology and Neurosurgery Recruiting
London, United Kingdom, WC1N 3BG
Contact: Ana Amado Fondo       AnaAmado.Fondo@uclh.nhs.uk   
Principal Investigator: Elaine Murphy, MD         
Sub-Investigator: Robin Lachmann, MD         
Great Ormond Street Hospital NHS Foundation Trust Recruiting
London, United Kingdom, WC1N 6NH
Contact: Ali Hamida    0207-405-9200 ext 6894    hamida.ali@gosh.nhs.uk   
Principal Investigator: Maureen Cleary, MD         
St. Mary's Hospital Recruiting
Manchester, United Kingdom, M13 9WL
Contact: Laura Crowther    +44 161 701 9137    elisabeth.jameson@cmft.nhs.uk   
Contact: Niamh Finnigan    +44 161 701 9259    niamh.finnigan@cmft.nhs.uk   
Principal Investigator: Elisabeth Jameson, MD         
Sub-Investigator: Simon Jones, MD         
Sub-Investigator: Alexander Broomfield, MD         
Salford Royal NHS Foundation Trust Recruiting
Salford, United Kingdom, M6 8HD
Contact: Marie Meehan    0161 206 4192    marie.meehan@srft.nhs.uk   
Principal Investigator: Karolina Stephien, MD         
Sub-Investigator: Reena Sharma, MD         
Sub-Investigator: Gisela Wilcox, MD         
Sub-Investigator: Ana Jovanovic, MD         
Sponsors and Collaborators
BioMarin Pharmaceutical
ICON plc
Investigators
Study Director: Medical Affairs Study Management

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT02294877     History of Changes
Other Study ID Numbers: 110-504
First Posted: November 19, 2014    Key Record Dates
Last Update Posted: August 17, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis IV
Osteochondrodysplasias
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases