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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02294877




Registration number
NCT02294877
Ethics application status
Date submitted
28/10/2014
Date registered
19/11/2014
Date last updated
5/03/2024

Titles & IDs
Public title
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Scientific title
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Secondary ID [1] 0 0
110-504
Universal Trial Number (UTN)
Trial acronym
MARS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mucopolysaccharidosis IV Type A 0 0
Morquio A Syndrome 0 0
MPS IVA 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Treatment: Drugs - Vimizim® (elosulfase alfa)

Treatment: Drugs: Vimizim® (elosulfase alfa)
Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS)
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety of Vimizim treated patients as measured by the incidences of Adverse Events, Serious Adverse Events, and immunology tests
Timepoint [1] 0 0
10 Years
Primary outcome [2] 0 0
Efficacy of Vimizim as measured by the 6MWT, 3MSCT, RFTs, FEV1, FIVC, FVC, and MVV
Timepoint [2] 0 0
10 years
Secondary outcome [1] 0 0
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
Timepoint [1] 0 0
10 years

Eligibility
Key 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
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/09/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
21/02/2024
Sample size
Target
Accrual to date
Final
418
Recruitment in Australia
Recruitment state(s)
NedlandsNSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Perth Children's Hospital - Perth
Recruitment hospital [2] 0 0
Children's Hospital of Westmead - Westmead
Recruitment hospital [3] 0 0
Westmead Hospital - Westmead
Recruitment hospital [4] 0 0
Children's Health Queensland Hospital and Health Service - South Brisbane
Recruitment hospital [5] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [6] 0 0
Royal Children's Hospital - Victoria - Parkville
Recruitment hospital [7] 0 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
6009 - Perth
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
- Westmead
Recruitment postcode(s) [4] 0 0
4101 - South Brisbane
Recruitment postcode(s) [5] 0 0
5000 - Adelaide
Recruitment postcode(s) [6] 0 0
3052 - Parkville
Recruitment postcode(s) [7] 0 0
6847 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Arkansas
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Delaware
Country [5] 0 0
United States of America
State/province [5] 0 0
District of Columbia
Country [6] 0 0
United States of America
State/province [6] 0 0
Florida
Country [7] 0 0
United States of America
State/province [7] 0 0
Georgia
Country [8] 0 0
United States of America
State/province [8] 0 0
Hawaii
Country [9] 0 0
United States of America
State/province [9] 0 0
Illinois
Country [10] 0 0
United States of America
State/province [10] 0 0
Iowa
Country [11] 0 0
United States of America
State/province [11] 0 0
Kentucky
Country [12] 0 0
United States of America
State/province [12] 0 0
Massachusetts
Country [13] 0 0
United States of America
State/province [13] 0 0
Michigan
Country [14] 0 0
United States of America
State/province [14] 0 0
Minnesota
Country [15] 0 0
United States of America
State/province [15] 0 0
Nevada
Country [16] 0 0
United States of America
State/province [16] 0 0
New Jersey
Country [17] 0 0
United States of America
State/province [17] 0 0
New York
Country [18] 0 0
United States of America
State/province [18] 0 0
North Carolina
Country [19] 0 0
United States of America
State/province [19] 0 0
Pennsylvania
Country [20] 0 0
United States of America
State/province [20] 0 0
Texas
Country [21] 0 0
United States of America
State/province [21] 0 0
Virginia
Country [22] 0 0
United States of America
State/province [22] 0 0
Washington
Country [23] 0 0
United States of America
State/province [23] 0 0
Wisconsin
Country [24] 0 0
Austria
State/province [24] 0 0
Graz
Country [25] 0 0
Austria
State/province [25] 0 0
Vienna
Country [26] 0 0
Belgium
State/province [26] 0 0
Edegem
Country [27] 0 0
Canada
State/province [27] 0 0
British Columbia
Country [28] 0 0
Canada
State/province [28] 0 0
Quebec
Country [29] 0 0
Czechia
State/province [29] 0 0
Prague
Country [30] 0 0
Denmark
State/province [30] 0 0
Dk-2100
Country [31] 0 0
France
State/province [31] 0 0
Bron Cedex
Country [32] 0 0
France
State/province [32] 0 0
Paris Cedex 15
Country [33] 0 0
France
State/province [33] 0 0
Vandœuvre-lès-Nancy
Country [34] 0 0
Ireland
State/province [34] 0 0
Dublin
Country [35] 0 0
Italy
State/province [35] 0 0
Catania
Country [36] 0 0
Italy
State/province [36] 0 0
Firenze
Country [37] 0 0
Italy
State/province [37] 0 0
Monza
Country [38] 0 0
Italy
State/province [38] 0 0
Naples
Country [39] 0 0
Malaysia
State/province [39] 0 0
Kuala Lumpur
Country [40] 0 0
Netherlands
State/province [40] 0 0
Amsterdam
Country [41] 0 0
Netherlands
State/province [41] 0 0
Rotterdam
Country [42] 0 0
Poland
State/province [42] 0 0
Warszawa
Country [43] 0 0
Portugal
State/province [43] 0 0
Coimbra
Country [44] 0 0
Portugal
State/province [44] 0 0
Lisboa
Country [45] 0 0
Portugal
State/province [45] 0 0
Lisbon
Country [46] 0 0
Puerto Rico
State/province [46] 0 0
San Juan
Country [47] 0 0
Taiwan
State/province [47] 0 0
Changhua County
Country [48] 0 0
Taiwan
State/province [48] 0 0
San-Min District
Country [49] 0 0
Taiwan
State/province [49] 0 0
Taoyuan County
Country [50] 0 0
Taiwan
State/province [50] 0 0
Kaohsiung City
Country [51] 0 0
Taiwan
State/province [51] 0 0
Kaohsiung
Country [52] 0 0
Taiwan
State/province [52] 0 0
Taichung
Country [53] 0 0
Taiwan
State/province [53] 0 0
Tainan
Country [54] 0 0
Taiwan
State/province [54] 0 0
Taipei
Country [55] 0 0
United Kingdom
State/province [55] 0 0
Belfast
Country [56] 0 0
United Kingdom
State/province [56] 0 0
Birmingham
Country [57] 0 0
United Kingdom
State/province [57] 0 0
London
Country [58] 0 0
United Kingdom
State/province [58] 0 0
Manchester
Country [59] 0 0
United Kingdom
State/province [59] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
BioMarin Pharmaceutical
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
ICON plc
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT02294877
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director, MD
Address 0 0
BioMarin Pharmaceutical
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries