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

study id #: NCT02294877

condition: Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA

status: Recruiting

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.

intervention: Vimizim® (elosulfase alfa)

results: https://clinicaltrials.gov/ct2/show/results/NCT02294877

last updated: March 05, 2022

start date: September 2014

estimated completion: September 2024

last updated: July 30, 2021

size / enrollment: 583

study 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.

primary outcomes:

• Safety of Vimizim treated patients as measured by the incidences of Adverse Events, Serious Adverse Events, and immunology tests
  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.
• 10 Years
  Efficacy of Vimizim as measured by the 6MWT, 3MSCT, RFTs, FEV1, FIVC, FVC, and MVV
  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.
• 10 years
  

secondary outcomes:

• 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
  10 years

inclusion 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: 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

sponsor: BioMarin Pharmaceutical

contacts: Trial Specialist, medinfo@bmrn.com

investigators: Medical Director, MD,BioMarin Pharmaceutical

trial center locations: United States,Australia,Austria,Belgium,Canada,Czechia,Denmark,France,Germany,Ireland,Italy,Malaysia,Netherlands,Poland,Portugal,Puerto Rico,Taiwan,United Kingdom

• United States, Arizona
  Phoenix Children’s Hospital
  
  
• United States, Arkansas
  Arkansas Children’s Hospital
  
  
• United States, California
  Children’s Hospital of Los Angeles
  
  
• United States, California
  Children’s Hospital of Orange County
  
  
• United States, California
  University of Southern California
  
  
• United States, Delaware
  A.I. Dupont Hospital for Children
  
  
• United States, District Of Columbia
  Children’s National Health System
  
  
• United States, Florida
  Miami Children’s Hospital
  
  
• United States, Georgia
  Emory University
  
  
• United States, Hawaii
  Kapiolani Medical Center for Women and Children
  
  
• United States, Illinois
  Ann & Robert H. Lurie Children’s Hospital
  
  
• United States, Iowa
  University Of Iowa
  
  
• United States, Kentucky
  University of Louisville Research Foundation Inc. – Kosair Charities Pediatric Clinical Research Unit
  
  
• United States, Massachusetts
  Children’s Hospital of Boston
  
  
• United States, Michigan
  University of Michigan
  
  
• United States, Minnesota
  Childrens Hospitals and Clinics of Minnesota
  
  
• United States, Nevada
  Children’s Specialty Center of Nevada
  
  
• United States, New Jersey
  Atlantic Health System
  
  
• United States, New Jersey
  St. Joseph’s Regional Medical Center
  
  
• United States, New York
  NYU-Langone Medical Center School of Medicine
  
  
• United States, North Carolina
  Duke University Medical Center
  
  
• United States, Pennsylvania
  Children’s Hospital of Philadelphia
  
  
• United States, Pennsylvania
  Children’s Hospital of Pittsburgh of UPMC
  
  
• United States, Texas
  UTHealth McGovern Medical School
  
  
• United States, Virginia
  Children’s Hospital of the King’s Daughters
  
  
• United States, Washington
  Seattle Children’s Hospital
  
  
• United States, Wisconsin
  Children’s Hospital of Wisconsin
  
  
• Australia, Nedlands
  Perth Children’s Hospital
  
  
• Australia, New South Wales
  Children’s Hospital of Westmead
  
  
• Australia, New South Wales
  Westmead Hospital
  
  
• Australia, Queensland
  Chidren’s Heatlh Queensland Hospital and Health Service
  
  
• Australia, South Australia
  Royal Adelaide Hospital
  
  
• Australia, Victoria
  Royal Children’s Hospital – Victoria
  
  
• Australia, Western Australia
  Royal Perth Hospital
  
  
• Austria
  Medical University of Vienna
  
  
• Austria
  Universitätsklinik Graz
  
  
• Belgium
  Antwerp University Hospital
  
  
• Canada, British Columbia
  Children’s & Women’s Centre of British Columbia
  
  
• Canada, Quebec
  McGill University Health Centre
  
  
• Canada, Quebec
  Sainte-Justine Hospital
  
  
• Canada, Quebec
  Université de Sherbrooke
  
  
• Czechia
  Klinika Dětského a Dorostového Lékařstvi
  
  
• Denmark, Dk-2100
  Copenhagen University Hospital, Klinik For Sjaeldne Handicap
  
  
• France
  Hopital d’enfants – Hopitaux de Brabois
  
  
• France
  Hospital Necker
  
  
• France
  Hôpital Femme Mère Enfant
  
  
• Germany
  Clinical Study Center, University Pediatric Clinic
  
  
• Germany
  University Medical Center, Johannes Gutenberg-University Mainz
  
  
• Germany
  Universitätsklinikum Hamburg-Eppendorf
  
  
• Germany
  Zentrum für seltene Erkrankungen, Horst-Schmidt-Kliniken
  
  
• Ireland
  Temple Street Children’s University Hospital
  
  
• Italy
  Azienda Ospedaliero Meyer
  
  
• Italy
  Azienda Ospedaliero San Gerardo di Monza
  
  
• Italy
  Azienda Policlinico Vittorio Emanuele, presidio G Rodolico, Università di Catania
  
  
• Italy
  Federico II University Hospital
  
  
• Malaysia
  Hospital Kuala Lumpur
  
  
• Malaysia
  University of Malay Medical Center
  
  
• Netherlands
  Academic Medical Center – University of Amsterdam
  
  
• Netherlands
  Rotterdam University Hospital Medical Centre
  
  
• Poland
  The Children Memorial Health Institute
  
  
• Portugal
  Centro Hospitalar Lisboa Centro – Hospital de Dona Estefânia
  
  
• Portugal
  Centro Hospitalar Lisboa Norte
  
  
• Portugal
  Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra)
  
  
• Puerto Rico
  San Jorge Children’s Hospital, Centro de Investigaciones Clinicas
  
  
• Taiwan
  Chang Gung Memorial Hospital Kaohsiung Branch
  
  
• Taiwan
  China Medical University and Hospital
  
  
• Taiwan
  Kaohsiung Veterans General Hospital
  
  
• Taiwan
  MacKay Memorial Hospital
  
  
• Taiwan
  National Cheng Kung University (NCKU) Hospital
  
  
• Taiwan
  National Taiwan University Hospital
  
  
• Taiwan
  Taichung Veterans General Hospital
  
  
• Taiwan, Changhua County
  Changhua Christian Hospital (CCH)
  
  
• Taiwan, San-Min District
  Kaohsiung Medical University Chung-Ho Memorial Hospital
  
  
• Taiwan, Taoyuan County
  Chang Gung Memorial Hospital
  
  
• United Kingdom
  Belfast City Hospital
  
  
• United Kingdom
  Birmingham Children’s Hospital
  
  
• United Kingdom
  Great Ormond Street Hospital NHS Foundation Trust
  
  
• United Kingdom
  National Hospital for Neurology and Neurosurgery
  
  
• United Kingdom
  Royal Free NHS Foundation Trust
  
  
• United Kingdom
  Royal Manchester Children’s Hospital – Manchester University NHS Foundation Trust
  
  
• United Kingdom
  Salford Royal NHS Foundation Trust
  
  
• United Kingdom
  University Hospital Birmingham – Selly Oaks