NCT02294877
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.
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.
All
Any
No
Vimizim® (elosulfase alfa)
Observational
418
2014-09
2024-03-05
Phoenix, Arizona, United States
Little Rock, Arkansas, United States
Los Angeles, California, United States
Los Angeles, California, United States
Orange, California, United States
Wilmington, Delaware, United States
Washington, District of Columbia, United States
Miami, Florida, United States
Decatur, Georgia, United States
Honolulu, Hawaii, United States
Chicago, Illinois, United States
Iowa City, Iowa, United States
Louisville, Kentucky, United States
Boston, Massachusetts, United States
Ann Arbor, Michigan, United States
Minneapolis, Minnesota, United States
Las Vegas, Nevada, United States
Morristown, New Jersey, United States
New York, New York, United States
Durham, North Carolina, United States
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
Houston, Texas, United States
Norfolk, Virginia, United States
Seattle, Washington, United States
Milwaukee, Wisconsin, United States
Perth, Nedlands, Australia
Westmead, New South Wales, Australia
Westmead, New South Wales, Australia
South Brisbane, Queensland, Australia
Adelaide, South Australia, Australia
Parkville, Victoria, Australia
Perth, Western Australia, Australia
Graz, , Austria
Vienna, , Austria
Edegem, , Belgium
Vancouver, British Columbia, Canada
Vancouver, British Columbia, Canada
Montreal, Quebec, Canada
Montreal, Quebec, Canada
Sherbrooke, Quebec, Canada
Prague, , Czechia
Copenhagen, Dk-2100, Denmark
Bron Cedex, , France
Paris Cedex 15, , France
Vandœuvre-lès-Nancy, , France
Dublin, , Ireland
Catania, , Italy
Firenze, , Italy
Monza, , Italy
Naples, , Italy
Kuala Lumpur, , Malaysia
Kuala Lumpur, , Malaysia
Amsterdam, , Netherlands
Rotterdam, , Netherlands
Warszawa, , Poland
Coimbra, , Portugal
Lisboa, , Portugal
Lisbon, , Portugal
San Juan, , Puerto Rico
Changhua City, Changhua County, Taiwan
Kaohsiung, San-Min District, Taiwan
Taoyuan City, Taoyuan County, Taiwan
Kaohsiung City, , Taiwan
Kaohsiung, , Taiwan
Taichung, , Taiwan
Taichung, , Taiwan
Tainan, , Taiwan
Taipei, , Taiwan
Taipei, , Taiwan
Taipei, , Taiwan
Belfast, , United Kingdom
Birmingham, , United Kingdom
Birmingham, , United Kingdom
London, , United Kingdom
London, , United Kingdom
London, , United Kingdom
Manchester, , United Kingdom
Salford, , United Kingdom
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:
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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