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

NCT02294877 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 418

Last updated 2024-03-05

No results posted yet for this study

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.

Conditions

  • Mucopolysaccharidosis IV Type A
  • Morquio A Syndrome
  • MPS IVA

Interventions

DRUG

Vimizim® (elosulfase alfa)

Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS)

Sponsors & Collaborators

Principal Investigators

  • Medical Director, MD · BioMarin Pharmaceutical

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-09-30
Primary Completion
2024-02-21
Completion
2024-02-21

Countries

  • United States
  • Australia
  • Austria
  • Belgium
  • Canada
  • Czechia
  • Denmark
  • France
  • Ireland
  • Italy
  • Malaysia
  • Netherlands
  • Poland
  • Portugal
  • Puerto Rico
  • Taiwan
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02294877 on ClinicalTrials.gov