Mucopolysaccharidosis IV Type AMARSNCT02294877
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
An observational study of Mucopolysaccharidosis IV Type A and Morquio A Syndrome, run by BioMarin Pharmaceutical at 79 sites in 17 countries.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- What is being tested?
- Vimizim® (elosulfase alfa)
- How many people
- 418 participants
- Where
- 79 sitesin 17 countries
- Study dates
- Sep 2014to 21 Feb 2024
- How long will I take part?
- 10 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with MPS IVA disease and patients treated with Vimizim, although patients are not required to receive Vimizim to be eligible to participate in this Registry.
Who may be able to join
- 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
Show all 13Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
79 sites in 17 countries.
79 sites in 17 countries
United States26 sites
University of Michigan
Ann Arbor, Michigan, 48109
Children's Hospital of Boston
Boston, Massachusetts, 02115
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611
Emory University
Decatur, Georgia, 30033
Duke University Medical Center
Durham, North Carolina, 27710
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96826
UTHealth McGovern Medical School
Houston, Texas, 77030
University Of Iowa
Iowa City, Iowa, 52524
Children's Specialty Center of Nevada
Las Vegas, Nevada, 89109
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Children's Hospital of Los Angeles
Los Angeles, California, 90027
University of Southern California
Los Angeles, California, 90033
University of Louisville Research Foundation Inc. - Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202
Miami Children's Hospital
Miami, Florida, 33155
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404
Atlantic Health System
Morristown, New Jersey, 07960
NYU-Langone Medical Center School of Medicine
New York, New York, 10016
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
Children's Hospital of Orange County
Orange, California, 92868
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Seattle Children's Hospital
Seattle, Washington, 98145-5005
Children's National Health System
Washington D.C., District of Columbia, 20010
A.I. Dupont Hospital for Children
Wilmington, Delaware, 19803
Taiwan11 sites
Changhua Christian Hospital (CCH)
Changhua, Changhua County, 50006
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, San-Min District, 80756
Kaohsiung Veterans General Hospital
Kaohsiung City, 81362
Chang Gung Memorial Hospital Kaohsiung Branch
Kaohsiung City, 83301
China Medical University and Hospital
Taichung, 40402
Taichung Veterans General Hospital
Taichung, 40705
National Cheng Kung University (NCKU) Hospital
Tainan, 70403
National Taiwan University Hospital
Taipei, 10050
MacKay Memorial Hospital
Taipei, 10449
Taipei Veterans General Hospital
Taipei, 11217
Chang Gung Memorial Hospital
Taoyuan City, Taoyuan County, 33305
United Kingdom8 sites
Belfast City Hospital
Belfast, BT9 7AB
University Hospital Birmingham - Selly Oaks
Birmingham, B15 2WB
Birmingham Children's Hospital
Birmingham, B4 6NH
Royal Free NHS Foundation Trust
London, NW3 2QG
National Hospital for Neurology and Neurosurgery
London, WC1N 3BG
Great Ormond Street Hospital NHS Foundation Trust
London, WC1N 3JH
Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust
Manchester, M13 9WL
Salford Royal NHS Foundation Trust
Salford, M6 8HD
Australia7 sites
Royal Adelaide Hospital
Adelaide, South Australia, 5000
Royal Children's Hospital - Victoria
Parkville, Victoria, 3052
Perth Children's Hospital
Perth, Nedlands, 6009
Royal Perth Hospital
Perth, Western Australia, 6847
Children's Health Queensland Hospital and Health Service
South Brisbane, Queensland, 4101
Children's Hospital of Westmead
Westmead, New South Wales, 2145
Westmead Hospital
Westmead, New South Wales
Canada5 sites
Sainte-Justine Hospital
Montreal, Quebec, H3T 1C5
McGill University Health Centre
Montreal, Quebec, H4A 3J1
Université de Sherbrooke
Sherbrooke, Quebec, J1H 5N4
Adult Metabolic Diseases Clinic Vancouver General Hospital
Vancouver, British Columbia, V5Z 1M9
Children's & Women's Centre of British Columbia
Vancouver, British Columbia, V6H 3N1
Italy4 sites
Azienda Policlinico Vittorio Emanuele, presidio G Rodolico, Università di Catania
Catania, 95125
Azienda Ospedaliero Meyer
Florence, 50139
Azienda Ospedaliero San Gerardo di Monza
Monza, 20090
Federico II University Hospital
Naples, 80131
France3 sites
Hôpital Femme Mère Enfant
Bron, 69677
Hospital Necker
Paris, 75743
Hopital d'enfants - Hopitaux de Brabois
Vandœuvre-lès-Nancy, 54500
Portugal3 sites
Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra)
Coimbra, 3000-062
Centro Hospitalar Lisboa Centro - Hospital de Dona Estefânia
Lisbon, 1169-045
Centro Hospitalar Lisboa Norte
Lisbon
Austria2 sites
Universitätsklinik Graz
Graz, 8036
Medical University of Vienna
Vienna, 1090
Malaysia2 sites
Hospital Kuala Lumpur
Kuala Lumpur, 50586
University of Malay Medical Center
Kuala Lumpur
Netherlands2 sites
Academic Medical Center - University of Amsterdam
Amsterdam, 1105 AZ
Rotterdam University Hospital Medical Centre
Rotterdam, 3015 GE
Denmark1 site
Copenhagen University Hospital, Klinik For Sjaeldne Handicap
Copenhagen, Dk-2100
Puerto Rico1 site
San Jorge Children's Hospital, Centro de Investigaciones Clinicas
San Juan, Commonwealth, 00912
About this study
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.
Read the detailed description
MARS is a multicenter, multinational, observational disease registry for patients diagnosed with Mucopolysaccharidosis Type IVA (MPS IVA). The Registry will collect medical history, and clinical and safety assessments every six months, for up to 10 years. 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 completed.
These assessments are designed to further characterize the spectrum of clinical signs and symptoms of the disease, and to further characterize the safety profile of Vimizim in a broader population. It is not required that patients receive Vimizim to be eligible to participate in this Registry.
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 clinical study or MOR-007 clinical study. After the 5 year period, these patients should remain in MARS until the Registry is complete.
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Being tested
- Vimizim® (elosulfase alfa)
- Medical subject headings
- Mucopolysaccharidosis IV
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vimizim® (elosulfase alfa)Drug
Also called BMN 110
Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS)
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of Vimizim treated patients as measured by the incidences of Adverse Events, Serious Adverse Events, and immunology tests
Additional safety measures are medications, occurrence of Bone Marrow Transplant/Hematopoietic Stem Cell Transplant, clinical laboratory tests, vital signs, Electrocardiograms, Echocardiograms, immunogenicity results, physical examinations, imaging studies, and cervical spine imaging.
All assessments in this observational study will be carried out per the participating institution's standard of care.
The MARS Annual report will descriptively summarize the safety and outcome measurements for the duration of this voluntary, observational study.
Measured: 10 Years
Efficacy of Vimizim as measured by the 6MWT, 3MSCT, RFTs, FEV1, FIVC, FVC, and MVV
Efficacy of Vimizim as measured by the changes in the 6 minute walk test (6MWT), 3 minute stir climb test (3MSCT), respiratory function tests (RFTs) including forced expiratory volume for 1 second (FEV1), forced inspiratory vital capacity (FIVC), forced vital capacity (FVC), and maximum voluntary ventilation (MVV) and by urinary Keratan Sulfate.
Measured: 10 years
Secondary outcomes (1)
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
The MARS Pregnancy Substudy will collect safety data on the outcomes of pregnancy in women who receive Vimizim as measured by Adverse Events, Serious Adverse Events, immunology tests, and the infant outcomes as measured by a live birth.
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2014 (estimated)
- Study startSep 2014
- Primary completion21 Feb 2024
- Study completion21 Feb 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis IV Type AStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 418 participants (actual)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02294877
- Sponsor's protocol ID
- 110-504
- Acronym
- MARS
- Status last verified
- Mar 2024
- First submitted
- 28 Oct 2014
- Last updated
- 5 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
- ClinicalTrials.gov ID
- NCT02294877
- Acronym / sponsor ID
- MARS / 110-504
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 418 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Study start
- Sep 2014
- Primary completion
- 21 Feb 2024 (actual)
- Study completion
- 21 Feb 2024 (actual)
- First posted
- 19 Nov 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
MARS is a multicenter, multinational, observational disease registry for patients diagnosed with Mucopolysaccharidosis Type IVA (MPS IVA). The Registry will collect medical history, and clinical and safety assessments every six months, for up to 10 years. 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 completed.
These assessments are designed to further characterize the spectrum of clinical signs and symptoms of the disease, and to further characterize the safety profile of Vimizim in a broader population. It is not required that patients receive Vimizim to be eligible to participate in this Registry.
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 clinical study or MOR-007 clinical study. After the 5 year period, these patients should remain in MARS until the Registry is complete.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with MPS IVA disease and patients treated with Vimizim, although patients are not required to receive Vimizim to be eligible to participate in this Registry.
Key inclusion criteria (13 listed)
- 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:
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vimizim® (elosulfase alfa)Also: BMN 110Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS) | Drug | – |
Primary outcome
Safety of Vimizim treated patients as measured by the incidences of Adverse Events, Serious Adverse Events, and immunology tests
Time frame: 10 Years
Additional safety measures are medications, occurrence of Bone Marrow Transplant/Hematopoietic Stem Cell Transplant, clinical laboratory tests, vital signs, Electrocardiograms, Echocardiograms, immunogenicity results, physical examinations, imaging studies, and cervical spine imaging. All assessments in this observational study will be carried out per the participating institution's standard of care. The MARS Annual report will descriptively summarize the safety and outcome measurements for the duration of this voluntary, observational study.
Efficacy of Vimizim as measured by the 6MWT, 3MSCT, RFTs, FEV1, FIVC, FVC, and MVV
Time frame: 10 years
Efficacy of Vimizim as measured by the changes in the 6 minute walk test (6MWT), 3 minute stir climb test (3MSCT), respiratory function tests (RFTs) including forced expiratory volume for 1 second (FEV1), forced inspiratory vital capacity (FIVC), forced vital capacity (FVC), and maximum voluntary ventilation (MVV) and by urinary Keratan Sulfate.
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director, MD study director
BioMarin Pharmaceutical
79 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mucopolysaccharidosis IV Type A and Morquio A Syndrome, run by BioMarin Pharmaceutical at 79 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 79 sites in United States, Taiwan, United Kingdom, Australia, Canada and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of Vimizim treated patients as measured by the incidences of Adverse Events, Serious Adverse Events, and immunology tests (time frame: 10 Years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2024.