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

Completed

Conditions studied: Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA

In brief

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.

Key facts

Study ID: NCT02294877
Run by: BioMarin Pharmaceutical
People needed: 418
Starts: 2014-09-01
Expected to finish: 2024-02-21
Last updated by the study team: 2024-03-05

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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

Where it is running

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 D.C., 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
Phoenix Children's Hospital — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.