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

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Trial Status Completed

Trial Identifier

NCT02294877

Condition

MPS IVA, Mucopolysaccharidosis IV Type A, Morquio A Syndrome

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

Any

Healthy Volunteers

No

Drug/Treatment:

Vimizim® (elosulfase alfa)

Study Type:

Observational

Number of Participants:

418

Study Started:

2014-09

Study Updated:

2024-03-05

Trial Locations

  • Phoenix Children's Hospital

    Phoenix, Arizona, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, United States

  • Children's Hospital of Los Angeles

    Los Angeles, California, United States

  • University of Southern California

    Los Angeles, California, United States

  • Children's Hospital of Orange County

    Orange, California, United States

  • A.I. Dupont Hospital for Children

    Wilmington, Delaware, United States

  • Children's National Health System

    Washington, District of Columbia, United States

  • Miami Children's Hospital

    Miami, Florida, United States

  • Emory University

    Decatur, Georgia, United States

  • Kapiolani Medical Center for Women and Children

    Honolulu, Hawaii, United States

  • Ann & Robert H. Lurie Children's Hospital

    Chicago, Illinois, United States

  • University Of Iowa

    Iowa City, Iowa, United States

  • University of Louisville Research Foundation Inc. - Kosair Charities Pediatric Clinical Research Unit

    Louisville, Kentucky, United States

  • Children's Hospital of Boston

    Boston, Massachusetts, United States

  • University of Michigan

    Ann Arbor, Michigan, United States

  • Children's Hospitals and Clinics of Minnesota

    Minneapolis, Minnesota, United States

  • Children's Specialty Center of Nevada

    Las Vegas, Nevada, United States

  • Atlantic Health System

    Morristown, New Jersey, United States

  • NYU-Langone Medical Center School of Medicine

    New York, New York, United States

  • Duke University Medical Center

    Durham, North Carolina, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, United States

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, United States

  • UTHealth McGovern Medical School

    Houston, Texas, United States

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, United States

  • Seattle Children's Hospital

    Seattle, Washington, United States

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, United States

  • Perth Children's Hospital

    Perth, Nedlands, Australia

  • Children's Hospital of Westmead

    Westmead, New South Wales, Australia

  • Westmead Hospital

    Westmead, New South Wales, Australia

  • Children's Health Queensland Hospital and Health Service

    South Brisbane, Queensland, Australia

  • Royal Adelaide Hospital

    Adelaide, South Australia, Australia

  • Royal Children's Hospital - Victoria

    Parkville, Victoria, Australia

  • Royal Perth Hospital

    Perth, Western Australia, Australia

  • Universitätsklinik Graz

    Graz, , Austria

  • Medical University of Vienna

    Vienna, , Austria

  • Antwerp University Hospital

    Edegem, , Belgium

  • Adult Metabolic Diseases Clinic Vancouver General Hospital

    Vancouver, British Columbia, Canada

  • Children's & Women's Centre of British Columbia

    Vancouver, British Columbia, Canada

  • Sainte-Justine Hospital

    Montreal, Quebec, Canada

  • McGill University Health Centre

    Montreal, Quebec, Canada

  • Université de Sherbrooke

    Sherbrooke, Quebec, Canada

  • Klinika Dětského a Dorostového Lékařstvi

    Prague, , Czechia

  • Copenhagen University Hospital, Klinik For Sjaeldne Handicap

    Copenhagen, Dk-2100, Denmark

  • Hôpital Femme Mère Enfant

    Bron Cedex, , France

  • Hospital Necker

    Paris Cedex 15, , France

  • Hopital d'enfants - Hopitaux de Brabois

    Vandœuvre-lès-Nancy, , France

  • Temple Street Children's University Hospital

    Dublin, , Ireland

  • Azienda Policlinico Vittorio Emanuele, presidio G Rodolico, Università di Catania

    Catania, , Italy

  • Azienda Ospedaliero Meyer

    Firenze, , Italy

  • Azienda Ospedaliero San Gerardo di Monza

    Monza, , Italy

  • Federico II University Hospital

    Naples, , Italy

  • Hospital Kuala Lumpur

    Kuala Lumpur, , Malaysia

  • University of Malay Medical Center

    Kuala Lumpur, , Malaysia

  • Academic Medical Center - University of Amsterdam

    Amsterdam, , Netherlands

  • Rotterdam University Hospital Medical Centre

    Rotterdam, , Netherlands

  • The Children Memorial Health Institute

    Warszawa, , Poland

  • Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra)

    Coimbra, , Portugal

  • Centro Hospitalar Lisboa Norte

    Lisboa, , Portugal

  • Centro Hospitalar Lisboa Centro - Hospital de Dona Estefânia

    Lisbon, , Portugal

  • San Jorge Children's Hospital, Centro de Investigaciones Clinicas

    San Juan, , Puerto Rico

  • Changhua Christian Hospital (CCH)

    Changhua City, Changhua County, Taiwan

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung, San-Min District, Taiwan

  • Chang Gung Memorial Hospital

    Taoyuan City, Taoyuan County, Taiwan

  • Chang Gung Memorial Hospital Kaohsiung Branch

    Kaohsiung City, , Taiwan

  • Kaohsiung Veterans General Hospital

    Kaohsiung, , Taiwan

  • China Medical University and Hospital

    Taichung, , Taiwan

  • Taichung Veterans General Hospital

    Taichung, , Taiwan

  • National Cheng Kung University (NCKU) Hospital

    Tainan, , Taiwan

  • National Taiwan University Hospital

    Taipei, , Taiwan

  • MacKay Memorial Hospital

    Taipei, , Taiwan

  • Taipei Veterans General Hospital

    Taipei, , Taiwan

  • Belfast City Hospital

    Belfast, , United Kingdom

  • University Hospital Birmingham - Selly Oaks

    Birmingham, , United Kingdom

  • Birmingham Children's Hospital

    Birmingham, , United Kingdom

  • Royal Free NHS Foundation Trust

    London, , United Kingdom

  • National Hospital for Neurology and Neurosurgery

    London, , United Kingdom

  • Great Ormond Street Hospital NHS Foundation Trust

    London, , United Kingdom

  • Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust

    Manchester, , United Kingdom

  • Salford Royal NHS Foundation Trust

    Salford, , United Kingdom

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 tria

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