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

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.
 
ClinicalTrials.gov Identifier: NCT02294877
Recruitment Status : Recruiting
First Posted : November 19, 2014
Last Update Posted : October 26, 2020
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.

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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.


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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
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Contact: Trial Specialist medinfo@bmrn.com

Locations
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United States, Arizona
Phoenix Children's Hospital Not yet recruiting
Phoenix, Arizona, United States, 85016
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
United States, California
Children's Hospital Los Angeles Not yet recruiting
Los Angeles, California, United States, 90027
University of Southern California Recruiting
Los Angeles, California, United States, 90033
Children's Hospital of Orange County Recruiting
Orange, California, United States, 92868
United States, Delaware
A.I. Dupont Hospital for Children Recruiting
Wilmington, Delaware, United States, 19803
United States, District of Columbia
Children's National Health System Recruiting
Washington, District of Columbia, United States, 20010
United States, Florida
Miami Children's Hospital Recruiting
Miami, Florida, United States, 33155
United States, Georgia
Emory University Recruiting
Decatur, Georgia, United States, 30033
United States, Hawaii
Kapiolani Medical Center for Women and Children Recruiting
Honolulu, Hawaii, United States, 96826
United States, Illinois
Ann & Robert H. Lurie Children's Hospital Recruiting
Chicago, Illinois, United States, 60611
United States, Iowa
University Of Iowa Recruiting
Iowa City, Iowa, United States, 52524
United States, Kentucky
University of Louisville Research Foundation Inc. - Kosair Charities Pediatric Clinical Research Unit Terminated
Louisville, Kentucky, United States, 40202
United States, Massachusetts
Children's Hospital of Boston Recruiting
Boston, Massachusetts, United States, 02115
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
Childrens Hospitals and Clinics of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55404
United States, Nevada
Children's Specialty Center of Nevada Recruiting
Las Vegas, Nevada, United States, 89109
United States, New Jersey
Atlantic Health System Recruiting
Morristown, New Jersey, United States, 07960
St. Joseph's Regional Medical Center Not yet recruiting
Paterson, New Jersey, United States, 07503
United States, New York
NYU-Langone Medical Center School of Medicine Recruiting
New York, New York, United States, 10016
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
UTHealth McGovern Medical School Not yet recruiting
Houston, Texas, United States, 77030
United States, Virginia
Children's Hospital of the King's Daughters Recruiting
Norfolk, Virginia, United States, 23507
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98145-5005
United States, Wisconsin
Children's Hospital of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Australia, Nedlands
Government Of Western Australia - Princess Margaret Hospital For Children (PMH) Recruiting
Perth, Nedlands, Australia, 6009
Australia, New South Wales
Children's Hospital of Westmead Recruiting
Westmead, New South Wales, Australia, 2145
Westmead Hospital Not yet recruiting
Westmead, New South Wales, Australia
Australia, Queensland
Chidren's Heatlh Queensland Hospital and Health Service Recruiting
South Brisbane, Queensland, Australia, 4101
Australia, South Australia
Royal Adelaide Hospital Recruiting
Adelaide, South Australia, Australia, 5000
Australia, Victoria
Royal Children's Hospital - Victoria Recruiting
Parkville, Victoria, Australia, 3052
Australia, Western Australia
Royal Perth Hospital Not yet recruiting
Perth, Western Australia, Australia, 6847
Austria
Medical University of Vienna Recruiting
Vienna, Austria, 1090
Belgium
Antwerp University Hospital Recruiting
Edegem, Belgium, 2650
Canada, British Columbia
Children's & Women's Centre of British Columbia Recruiting
Vancouver, British Columbia, Canada, V6H 3N1
Canada, Quebec
Sainte-Justine Hospital Recruiting
Montreal, Quebec, Canada, H3T 1C5
McGill University Health Centre Recruiting
Montreal, Quebec, Canada, H4A 3J1
Université de Sherbrooke Recruiting
Sherbrooke, Quebec, Canada, J1H 5N4
Czechia
Klinika Dětského a Dorostového Lékařstvi Recruiting
Prague, Czechia, 12000
Denmark
Copenhagen University Hospital, Klinik For Sjaeldne Handicap Recruiting
Copenhagen, Dk-2100, Denmark
France
Hôpital Femme Mère Enfant Recruiting
Bron Cedex, France, 69677
Hopital Necker Not yet recruiting
Paris Cedex 15, France, 75743
Hopital d'enfants - Hopitaux de Brabois Recruiting
Vandœuvre-lès-Nancy, France, 54500
Germany
Clinical Study Center, University Pediatric Clinic Not yet recruiting
Bochum, Germany, 44791
Universitätsklinikum Hamburg-Eppendorf Not yet recruiting
Hamburg, Germany, 20246
University Medical Center, Johannes Gutenberg-University Mainz Not yet recruiting
Mainz, Germany, 55131
Zentrum für seltene Erkrankungen, Horst-Schmidt-Kliniken Not yet recruiting
Wiesbaden, Germany, 65199
Ireland
Temple Street Children's University Hospital Recruiting
Dublin, Ireland
Italy
Azienda Policlinico Vittorio Emanuele, presidio G Rodolico, Università di Catania Recruiting
Catania, Italy, 95125
Azienda Ospedaliero Meyer Recruiting
Firenze, Italy, 50139
Azienda Ospedaliero San Gerardo di Monza Recruiting
Monza, Italy, 20090
Federico II University Hospital Recruiting
Naples, Italy, 80131
Malaysia
Hospital Kuala Lumpur Recruiting
Kuala Lumpur, Malaysia, 50586
Netherlands
Academic Medical Center - University of Amsterdam Recruiting
Amsterdam, Netherlands, 1105 AZ
Rotterdam University Hospital Medical Centre Recruiting
Rotterdam, Netherlands, 3015 GE
Poland
The Children Memorial Health Institute Recruiting
Warszawa, Poland, 04-730
Portugal
Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra) Recruiting
Coimbra, Portugal, 3000-062
Centro Hospitalar Lisboa Norte Recruiting
Lisboa, Portugal
Centro Hospitalar Lisboa Centro - Hospital de Dona Estefânia Recruiting
Lisbon, Portugal, 1169-045
Puerto Rico
San Jorge Children's Hospital, Centro de Investigaciones Clinicas Recruiting
San Juan, Puerto Rico, 00912
Taiwan
Changhua Christian Hospital (CCH) Recruiting
Changhua City, Changhua County, Taiwan, 50006
Kaohsiung Medical University Chung-Ho Memorial Hospital Recruiting
Kaohsiung, San-Min District, Taiwan, 80756
Chang Gung Memorial Hospital Recruiting
Taoyuan City, Taoyuan County, Taiwan, 33305
Chang Gung Memorial Hospital Kaohsiung Branch Recruiting
Kaohsiung City, Taiwan, 83301
Kaohsiung Veterans General Hospital Recruiting
Kaohsiung, Taiwan, 81362
China Medical University and Hospital Recruiting
Taichung, Taiwan, 40402
Taichung Veterans General Hospital Recruiting
Taichung, Taiwan, 40705
National Cheng Kung University (NCKU) Hospital Recruiting
Tainan, Taiwan, 70403
National Taiwan University Hospital Recruiting
Taipei, Taiwan, 10050
MacKay Memorial Hospital Recruiting
Taipei, Taiwan, 10449
United Kingdom
Belfast City Hospital Recruiting
Belfast, United Kingdom, BT9 7AB
University Hospital Birmingham - Selly Oaks Recruiting
Birmingham, United Kingdom, B15 2WB
Birmingham Children's Hospital Recruiting
Birmingham, United Kingdom, B4 6NH
Royal Free NHS Foundation Trust Recruiting
London, United Kingdom, NW3 2QG
National Hospital for Neurology and Neurosurgery Recruiting
London, United Kingdom, WC1N 3BG
Great Ormond Street Hospital NHS Foundation Trust Recruiting
London, United Kingdom, WC1N 3JH
Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust Recruiting
Manchester, United Kingdom, M13 9WL
Salford Royal NHS Foundation Trust Recruiting
Salford, United Kingdom, M6 8HD
Sponsors and Collaborators
BioMarin Pharmaceutical
ICON plc
Investigators
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Study Director: Medical Director, MD BioMarin Pharmaceutical
Additional Information:

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Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT02294877    
Other Study ID Numbers: 110-504
First Posted: November 19, 2014    Key Record Dates
Last Update Posted: October 26, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Osteochondrodysplasias
Mucopolysaccharidoses
Mucopolysaccharidosis IV
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