A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status:
Recruiting
Trial end date:
2024-09-01
Target enrollment:
Participant gender:
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.