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| Name | Class |
|---|---|
| ICON plc | INDUSTRY |
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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.
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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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vimizim® (elosulfase alfa) | Drug | Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS) |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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. |
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Inclusion Criteria:
Patients eligible to participate in this Registry must meet all of the following criteria:
Patients eligible to participate in the Registry Substudy for MOR-005 must meet all of the following criteria:
Patients eligible to participate in this Registry Substudy for MOR-007 must meet all of the following criteria:
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
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director, MD | BioMarin Pharmaceutical | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | United States | ||
| Arkansas Children's Hospital |
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Blood serum; urine samples
| 10 years |
| Little Rock |
| Arkansas |
| 72202 |
| United States |
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | United States |
| University of Southern California | Los Angeles | California | 90033 | United States |
| Children's Hospital of Orange County | Orange | California | 92868 | United States |
| A.I. Dupont Hospital for Children | Wilmington | Delaware | 19803 | United States |
| Children's National Health System | Washington D.C. | District of Columbia | 20010 | United States |
| Miami Children's Hospital | Miami | Florida | 33155 | United States |
| Emory University | Decatur | Georgia | 30033 | United States |
| Kapiolani Medical Center for Women and Children | Honolulu | Hawaii | 96826 | United States |
| Ann & Robert H. Lurie Children's Hospital | Chicago | Illinois | 60611 | United States |
| University Of Iowa | Iowa City | Iowa | 52524 | United States |
| University of Louisville Research Foundation Inc. - Kosair Charities Pediatric Clinical Research Unit | Louisville | Kentucky | 40202 | United States |
| Children's Hospital of Boston | Boston | Massachusetts | 02115 | United States |
| University of Michigan | Ann Arbor | Michigan | 48109 | United States |
| Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota | 55404 | United States |
| Children's Specialty Center of Nevada | Las Vegas | Nevada | 89109 | United States |
| Atlantic Health System | Morristown | New Jersey | 07960 | United States |
| NYU-Langone Medical Center School of Medicine | New York | New York | 10016 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| UTHealth McGovern Medical School | Houston | Texas | 77030 | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23507 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98145-5005 | United States |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| Perth Children's Hospital | Perth | Nedlands | 6009 | Australia |
| Children's Hospital of Westmead | Westmead | New South Wales | 2145 | Australia |
| Westmead Hospital | Westmead | New South Wales | Australia |
| Children's Health Queensland Hospital and Health Service | South Brisbane | Queensland | 4101 | Australia |
| Royal Adelaide Hospital | Adelaide | South Australia | 5000 | Australia |
| Royal Children's Hospital - Victoria | Parkville | Victoria | 3052 | Australia |
| Royal Perth Hospital | Perth | Western Australia | 6847 | Australia |
| Universitätsklinik Graz | Graz | 8036 | Austria |
| Medical University of Vienna | Vienna | 1090 | Austria |
| Antwerp University Hospital | Edegem | 2650 | Belgium |
| Adult Metabolic Diseases Clinic Vancouver General Hospital | Vancouver | British Columbia | V5Z 1M9 | Canada |
| Children's & Women's Centre of British Columbia | Vancouver | British Columbia | V6H 3N1 | Canada |
| Sainte-Justine Hospital | Montreal | Quebec | H3T 1C5 | Canada |
| McGill University Health Centre | Montreal | Quebec | H4A 3J1 | Canada |
| Université de Sherbrooke | Sherbrooke | Quebec | J1H 5N4 | Canada |
| Klinika Dětského a Dorostového Lékařstvi | Prague | 12000 | Czechia |
| Copenhagen University Hospital, Klinik For Sjaeldne Handicap | Copenhagen | Dk-2100 | Denmark |
| Hôpital Femme Mère Enfant | Bron | 69677 | France |
| Hospital Necker | Paris | 75743 | France |
| Hopital d'enfants - Hopitaux de Brabois | Vandœuvre-lès-Nancy | 54500 | France |
| Temple Street Children's University Hospital | Dublin | Ireland |
| Azienda Policlinico Vittorio Emanuele, presidio G Rodolico, Università di Catania | Catania | 95125 | Italy |
| Azienda Ospedaliero Meyer | Florence | 50139 | Italy |
| Azienda Ospedaliero San Gerardo di Monza | Monza | 20090 | Italy |
| Federico II University Hospital | Naples | 80131 | Italy |
| Hospital Kuala Lumpur | Kuala Lumpur | 50586 | Malaysia |
| University of Malay Medical Center | Kuala Lumpur | Malaysia |
| Academic Medical Center - University of Amsterdam | Amsterdam | 1105 AZ | Netherlands |
| Rotterdam University Hospital Medical Centre | Rotterdam | 3015 GE | Netherlands |
| The Children Memorial Health Institute | Warsaw | 04-730 | Poland |
| Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra) | Coimbra | 3000-062 | Portugal |
| Centro Hospitalar Lisboa Centro - Hospital de Dona Estefânia | Lisbon | 1169-045 | Portugal |
| Centro Hospitalar Lisboa Norte | Lisbon | Portugal |
| San Jorge Children's Hospital, Centro de Investigaciones Clinicas | San Juan | Commonwealth | 00912 | Puerto Rico |
| Changhua Christian Hospital (CCH) | Changhua | Changhua County | 50006 | Taiwan |
| Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung City | San-Min District | 80756 | Taiwan |
| Chang Gung Memorial Hospital | Taoyuan City | Taoyuan County | 33305 | Taiwan |
| Kaohsiung Veterans General Hospital | Kaohsiung City | 81362 | Taiwan |
| Chang Gung Memorial Hospital Kaohsiung Branch | Kaohsiung City | 83301 | Taiwan |
| China Medical University and Hospital | Taichung | 40402 | Taiwan |
| Taichung Veterans General Hospital | Taichung | 40705 | Taiwan |
| National Cheng Kung University (NCKU) Hospital | Tainan | 70403 | Taiwan |
| National Taiwan University Hospital | Taipei | 10050 | Taiwan |
| MacKay Memorial Hospital | Taipei | 10449 | Taiwan |
| Taipei Veterans General Hospital | Taipei | 11217 | Taiwan |
| Belfast City Hospital | Belfast | BT9 7AB | United Kingdom |
| University Hospital Birmingham - Selly Oaks | Birmingham | B15 2WB | United Kingdom |
| Birmingham Children's Hospital | Birmingham | B4 6NH | United Kingdom |
| Royal Free NHS Foundation Trust | London | NW3 2QG | United Kingdom |
| National Hospital for Neurology and Neurosurgery | London | WC1N 3BG | United Kingdom |
| Great Ormond Street Hospital NHS Foundation Trust | London | WC1N 3JH | United Kingdom |
| Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust | Manchester | M13 9WL | United Kingdom |
| Salford Royal NHS Foundation Trust | Salford | M6 8HD | United Kingdom |
| ID | Term |
|---|---|
| D009085 | Mucopolysaccharidosis IV |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C102295 | GALNS protein, human |
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