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

• Register: ClinicalTrials.gov
• Last refreshed on: 18 March 2024
• Main ID: NCT02294877
• Date of registration: 28/10/2014
• Prospective Registration: No
• Primary sponsor: BioMarin Pharmaceutical
• Public title: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) MARS
• Scientific title: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
• Date of first enrolment: September 2014
• Target sample size: 418
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02294877
• Study type: Observational [Patient Registry]

Countries of recruitment

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

Contacts

• Name: Medical Director, MD
• Affiliation: BioMarin Pharmaceutical

Key inclusion & exclusion criteria

Inclusion Criteria:

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

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



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis IV Type A

Morquio A Syndrome

MPS IVA

Intervention(s)

Drug: Vimizim® (elosulfase alfa)

Primary Outcome(s)

Safety of Vimizim treated patients as measured by the incidences of Adverse Events, Serious Adverse Events, and immunology tests [Time Frame: 10 Years]

Efficacy of Vimizim as measured by the 6MWT, 3MSCT, RFTs, FEV1, FIVC, FVC, and MVV [Time Frame: 10 years]

Secondary Outcome(s)

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 [Time Frame: 10 years]

Secondary ID(s)

110-504

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

ICON plc