Study identification

EU PAS number

EUPAS6835

Study ID

49863

Official title and acronym

Morquio A Registry Study (MARS)

DARWIN EU® study

No

Study countries

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

Study description

A multicenter, multinational, observational Morquio A Registry Study (MARS) will be established to characterize and describe the MPS IVA population as a whole, including the heterogeneity, progression, and natural history of MPS IVA and to track the clinical outcomes of patients with MPS IVA treated with Vimizim®. The Registry will enroll and collect data on patients over a period of at least 8 years from the time of the first marketing approval globally and data on individual patients will continue to be collected for at least 2 years from the time the last patient was enrolled or until the Registry is terminated. It is not required that patients receive Vimizim to be eligible to participate in this Registry, however, they must have confirmed diagnosis of MPS IVA. Patients currently participating in a BMN 110 (elosulfase alfa) clinical trial will not meet inclusion criteria, but will be able to enroll after withdrawal from the clinical trial. The Registry will collect medical history, clinical, and safety assessments at least every six months or as indicated in the Recommended Schedule of Events, for up to 10 years. In addition this Registry will collect additional data on patients who have completed the MOR-005 and MOR-007 clinical trials. The MOR-005 and MOR-007 clinical trial patients will be enrolled into the appropriate Registry Substudy for a minimum of 5 years from the time of the patient's enrollment in the MOR-005 or MOR-007 clinical study. After the 5-year period, these patients should remain in MARS until the Registry is complete. Relevant retrospective data may also be collected. Registry data collected using a validated web-based application will be analyzed as per the Registry’s statistical analysis plan (SAP) and reported annually. The Morquio A Registry Study (MARS) will provide the necessary data to further characterize the spectrum of clinical signs and symptoms of the disease, and to further characterize the safety profile of Vimizim.

Study status

Ongoing

Contact details

Director Program

Primary lead investigator

Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Data analysis start date

Actual:

Date of interim report, if expected

Planned:
Actual:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

BioMarin International Limited
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

EU RMP category 1 (imposed as condition of marketing authorisation)