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

This study is currently recruiting participants.
Verified January 2017 by BioMarin Pharmaceutical
Sponsor:
ClinicalTrials.gov Identifier:
NCT02294877
First Posted: November 19, 2014
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.
Collaborator:
ICON plc
Information provided by (Responsible Party):
BioMarin Pharmaceutical
  Purpose
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 Intervention
Mucopolysaccharidosis IV Type A Morquio A Syndrome MPS IVA Drug: Vimizim® (elosulfase alfa)

Study Type: Observational [Patient Registry]
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 10 Years
Official Title: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

Resource links provided by NLM:


Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • 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.


  • 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:
  • 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

Estimated Enrollment: 583
Study Start Date: September 2014
Estimated Study Completion Date: September 2024
Estimated Primary Completion Date: September 2024 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Vimizim® (elosulfase alfa)
    Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS)
    Other Name: BMN 110
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.

  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
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

  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): NCT02294877


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

  Hide Study Locations
Locations
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Contact: Patricia Brady    501-364-1544    bradypatriciam@uams.edu   
Principal Investigator: Adolfo Garnica, 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   
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         
United States, District of Columbia
Children's National Health System Recruiting
Washington, D.C., District of Columbia, United States, 20010
Contact: Katie Crosby, CGC    202-476-6812    kacrosby@childrensnational.org   
Contact: Lindsay Kehoe, CGC    (202) 476-4549    lkehoe@childrensnational.org   
Principal Investigator: Pranoot Tanpaiboon, MD         
United States, Florida
Miami Children's Hospital Recruiting
Miami, Florida, United States, 33155
Contact: Yasameen Kharazmi    786-268-1741    yazameen.kharazmi@mch.com   
Principal Investigator: Mislen Bauer, MD         
United States, Georgia
Suma Shankar, MD Recruiting
Decatur, Georgia, United States, 30033
Contact: Stephanie Cagle    404-778-8618    scagle@emory.edu   
Principal Investigator: Suma Shankar, 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   
Contact: Karen Uehara    800-562-4789    karen.uehara@hawaiipacifichealth.org   
Principal Investigator: Laurie Seaver, MD         
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         
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   
Principal Investigator: Gordon Gowans, MD         
Sub-Investigator: Janice Sullivan, MD         
Sub-Investigator: Kara Goodin, MD         
Sub-Investigator: Alexander Asamoah, MD         
United States, Massachusetts
Children's Hospital of Boston Recruiting
Boston, Massachusetts, United States, 02115
Contact: Amy Kritzer, MD    857-218-4636    amy.kritzer@childrens.harvard.edu   
Principal Investigator: Amy Kritzer, MD         
United States, Minnesota
Childrens Hospitals and Clinics of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55404
Contact: Lisa Read    612-813-6658    lisa.read@childrensmn.org   
Contact: Molly Barrett    651-220-5730    molly.barrett@childrensmn.org   
Principal Investigator: Vikas Bhambani, MD         
Sub-Investigator: Nancy J. Mendelsohn, MD         
Sub-Investigator: Rebecca Olson, RN,CNP,APNG         
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, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Emma Bedoukian, CGC    215-590-3692    bedoukiane@email.chop.edu   
Contact: Ellen Purpus    (215) 590-4660    purpus@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: Areeg El-Gharbawy, MD         
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: Elizabeth Neumeister-Chisholm, MS         
Sub-Investigator: Katherine G Langley, MS         
Sub-Investigator: Angela M Casey, RN         
Sub-Investigator: Brooke Blessing-Spangler, MS         
Sub-Investigator: Benjamin M Helm, MS         
Sub-Investigator: A. Corina Moorehead, LPN, BS         
Sub-Investigator: Carrie E Mishoe, 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         
Austria
Universitätsklinik Graz Recruiting
Graz, Austria, A-8036
Contact: Michaela Brunner-Krainz, MD    43-316-385-2813    michaela.brunner@klinikum-graz.at   
Principal Investigator: Michaela Brunner-Krainz, MD         
Vassiliki Konstantopoulo, MD Recruiting
Vienna, Austria, 1090
Contact: Vassiliki Konstantopoulo, MD    0043 40400-32320    Vassiliki.konstantopoulo@meduniwien.ac.at   
Principal Investigator: Vassiliki Konstantopoulo, MD         
Belgium
Francois Eyskens, MD 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
McGill University Health Centre Recruiting
Montreal, Quebec, Canada, H4A 3J1
Contact: Sandra Pepin    514-9341934 ext 23737    sandra.pepin@muhc.mcgill.ca   
Principal Investigator: John Mitchell, MD         
Université de Sherbrooke Recruiting
Sherbrooke, Quebec, Canada, J1H 5N4
Contact: Caroline Barr    819-346-1110 ext 13933    cbarr.chus@ssss.gouv.qc.ca   
Principal Investigator: Bruno Maranda, MD         
Denmark
Klinik For Sjaeldne Handicap Recruiting
Copenhagen, Dk-2100, Denmark
Contact: Pernille Stroem    45 21120336    pernille.stroem@regionh.dk   
Principal Investigator: Christine Dali, MD         
Germany
Clinical Study Center, University Pediatric Clinic Recruiting
Bochum, Germany, 4791
Contact: Sandra Boeger    0234-509 2611    s.boeger@klinikum-Bochum.de   
Principal Investigator: Nesrin Karabul, MD         
Ireland
Belfast City Hospital Recruiting
Belfast, Ireland, BT9 7AB
Contact: Alison Murphy    02890636366    alison.murphy@belfasttrust.hscni.net   
Principal Investigator: Fiona Stewart, MD         
Temple Street Children's University Hospital Recruiting
Dublin, Ireland
Contact: Eithne Losty    01 8921903    ellen.crushell@cuh.ie   
Principal Investigator: Ahmad Monavari, MD         
Sub-Investigator: Ellen Crushell, MD         
Sub-Investigator: Ina Knerr, MD         
Sub-Investigator: Josephine Hughes, MD         
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 Kerr, 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         
Azienda Ospedaliero Meyer Recruiting
Firenze, Italy, 50132
Contact: Alice Donati, MD    0039 055 5662482    m.donati@meyer.it   
Principal Investigator: Alice Donati, 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         
Federico II University Hospital Recruiting
Naples, Italy, 80131
Contact: Roberto Della Casa, MD    390817462673    roberto.dellacasa@unin.it   
Principal Investigator: Roberto Della Casa, MD         
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         
Ana Van der Ploeg, MD Recruiting
Rotterdam, Netherlands, 3015 GE
Contact: Ana Van der Ploeg, MD    31 107032083      
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      
Principal Investigator: Anna Tylki-Szymanska, MD         
Portugal
Centro Hospitalar Lisboa Centro - Hospital de Dona Estefânia Recruiting
Rua Jacinta Marto, Lisbon, Portugal, 1169-045
Contact: Silvia Sequeira, MD    +351 21 312 68 94    silviasequeira@gmail.com   
Principal Investigator: Silvia Sequeira, MD         
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         
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         
Puerto Rico
San Jorge Children's Hospital Recruiting
San Juan, Puerto Rico, 00912
Contact: Heriberto Roman-Berrios    787-999-9453    heriberto.roman@sanjorgechildrenshospital.com   
Principal Investigator: Juan A Santiago-Cornier, MD         
United Kingdom
Birmingham Children's Hospital Recruiting
Birmingham, United Kingdom, B4 6NH
Contact: Alice Stewart    0121 333 9907    Alice.stewart@bch.nhs.uk   
Principal Investigator: Saikat Santra, MA/MB/BCh         
Sub-Investigator: Suresh Vijayaraghavan, MB         
Queen Elizabeth Hospital Birmingham Recruiting
Edgbaston, Birmingham, United Kingdom, B15 2WB
Contact: Shaun Bolton    44121-371-6795    shaun.bolton@uhb.nhs.uk   
Principal Investigator: Tarekegn G Hiwot, MD         
Sub-Investigator: Charlotte Dawson, MD         
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         
University College London Hospitals NHS Foundation Trust Recruiting
London, United Kingdom, WC1N 3BG
Contact: Roman Jastrub    0203 448 3607    roman.jastrub@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: Ruth Brodie    0207-405-9200 ext 6894    ruth.brodie@gosh.nhs.uk   
Principal Investigator: Maureen Cleary, MD         
St. Mary's Hospital Recruiting
Manchester, United Kingdom, M13 9WL
Contact: Jean Mercer, MBBCH, MRCPCH    0044 161 7012138    elisabeth.jameson@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 Greater Manchester, United Kingdom, M6 8HD
Contact: Marie Meehan    0161 206 4192    marie.meehan@srft.nhs.uk   
Principal Investigator: Christian Hendriksz, MD         
Sub-Investigator: Reena Sharma, MD         
Sub-Investigator: Gisela Wilcox, MD         
Sub-Investigator: Ana Jovanovic, MD         
Sponsors and Collaborators
BioMarin Pharmaceutical
ICON plc
  More Information

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT02294877     History of Changes
Other Study ID Numbers: 110-504
First Submitted: October 28, 2014
First Posted: November 19, 2014
Last Update Posted: April 21, 2017
Last Verified: January 2017
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