Clinical trial NCT02294877: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Official title: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Mucopolysaccharidosis IV Type A
Morquio A Syndrome
MPS IVA
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
Collaborators: name: ICON plc
First posted: 2014-11-19
Enrollment: 418
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

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.

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.

Eligibility 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

Primary outcomes:
measure: Safety of Vimizim treated patients as measured by the incidences of Adverse Events, Serious Adverse Events, and immunology tests
timeFrame: 10 Years
measure: Efficacy of Vimizim as measured by the 6MWT, 3MSCT, RFTs, FEV1, FIVC, FVC, and MVV
timeFrame: 10 years
Secondary outcomes:
measure: 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
timeFrame: 10 years
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: Los Angeles
country: United States
facility: University of Southern California
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: Wilmington
country: United States
facility: A.I. Dupont Hospital for Children
state: Delaware
city: Washington
country: United States
facility: Children's National Health System
state: District of Columbia
city: Miami
country: United States
facility: Miami Children's Hospital
state: Florida
city: Decatur
country: United States
facility: Emory University
state: Georgia
city: Honolulu
country: United States
facility: Kapiolani Medical Center for Women and Children
state: Hawaii
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital
state: Illinois
city: Iowa City
country: United States
facility: University Of Iowa
state: Iowa
city: Louisville
country: United States
facility: University of Louisville Research Foundation Inc. - Kosair Charities Pediatric Clinical Research Unit
state: Kentucky
city: Boston
country: United States
facility: Children's Hospital of Boston
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Minneapolis
country: United States
facility: Children's Hospitals and Clinics of Minnesota
state: Minnesota
city: Las Vegas
country: United States
facility: Children's Specialty Center of Nevada
state: Nevada
city: Morristown
country: United States
facility: Atlantic Health System
state: New Jersey
city: New York
country: United States
facility: NYU-Langone Medical Center School of Medicine
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
city: Houston
country: United States
facility: UTHealth McGovern Medical School
state: Texas
city: Norfolk
country: United States
facility: Children's Hospital of the King's Daughters
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
city: Perth
country: Australia
facility: Perth Children's Hospital
state: Nedlands
city: Westmead
country: Australia
facility: Children's Hospital of Westmead
state: New South Wales
city: Westmead
country: Australia
facility: Westmead Hospital
state: New South Wales
city: South Brisbane
country: Australia
facility: Children's Health Queensland Hospital and Health Service
state: Queensland
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
state: South Australia
city: Parkville
country: Australia
facility: Royal Children's Hospital - Victoria
state: Victoria
city: Perth
country: Australia
facility: Royal Perth Hospital
state: Western Australia
city: Graz
country: Austria
facility: Universitätsklinik Graz
city: Vienna
country: Austria
facility: Medical University of Vienna
city: Edegem
country: Belgium
facility: Antwerp University Hospital
city: Vancouver
country: Canada
facility: Adult Metabolic Diseases Clinic Vancouver General Hospital
state: British Columbia
city: Vancouver
country: Canada
facility: Children's & Women's Centre of British Columbia
state: British Columbia
city: Montreal
country: Canada
facility: Sainte-Justine Hospital
state: Quebec
city: Montreal
country: Canada
facility: McGill University Health Centre
state: Quebec
city: Sherbrooke
country: Canada
facility: Université de Sherbrooke
state: Quebec
city: Prague
country: Czechia
facility: Klinika Dětského a Dorostového Lékařstvi
city: Copenhagen
country: Denmark
facility: Copenhagen University Hospital, Klinik For Sjaeldne Handicap
state: Dk-2100
city: Bron Cedex
country: France
facility: Hôpital Femme Mère Enfant
city: Paris Cedex 15
country: France
facility: Hospital Necker
city: Vandœuvre-lès-Nancy
country: France
facility: Hopital d'enfants - Hopitaux de Brabois
city: Dublin
country: Ireland
facility: Temple Street Children's University Hospital
city: Catania
country: Italy
facility: Azienda Policlinico Vittorio Emanuele, presidio G Rodolico, Università di Catania
city: Firenze
country: Italy
facility: Azienda Ospedaliero Meyer
city: Monza
country: Italy
facility: Azienda Ospedaliero San Gerardo di Monza
city: Naples
country: Italy
facility: Federico II University Hospital
city: Kuala Lumpur
country: Malaysia
facility: Hospital Kuala Lumpur
city: Kuala Lumpur
country: Malaysia
facility: University of Malay Medical Center
city: Amsterdam
country: Netherlands
facility: Academic Medical Center - University of Amsterdam
city: Rotterdam
country: Netherlands
facility: Rotterdam University Hospital Medical Centre
city: Warszawa
country: Poland
facility: The Children Memorial Health Institute
city: Coimbra
country: Portugal
facility: Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra)
city: Lisboa
country: Portugal
facility: Centro Hospitalar Lisboa Norte
city: Lisbon
country: Portugal
facility: Centro Hospitalar Lisboa Centro - Hospital de Dona Estefânia
city: San Juan
country: Puerto Rico
facility: San Jorge Children's Hospital, Centro de Investigaciones Clinicas
city: Changhua City
country: Taiwan
facility: Changhua Christian Hospital (CCH)
state: Changhua County
city: Kaohsiung
country: Taiwan
facility: Kaohsiung Medical University Chung-Ho Memorial Hospital
state: San-Min District
city: Taoyuan City
country: Taiwan
facility: Chang Gung Memorial Hospital
state: Taoyuan County
city: Kaohsiung City
country: Taiwan
facility: Chang Gung Memorial Hospital Kaohsiung Branch
city: Kaohsiung
country: Taiwan
facility: Kaohsiung Veterans General Hospital
city: Taichung
country: Taiwan
facility: China Medical University and Hospital
city: Taichung
country: Taiwan
facility: Taichung Veterans General Hospital
city: Tainan
country: Taiwan
facility: National Cheng Kung University (NCKU) Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Taipei
country: Taiwan
facility: MacKay Memorial Hospital
city: Taipei
country: Taiwan
facility: Taipei Veterans General Hospital
city: Belfast
country: United Kingdom
facility: Belfast City Hospital
city: Birmingham
country: United Kingdom
facility: University Hospital Birmingham - Selly Oaks
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: London
country: United Kingdom
facility: Royal Free NHS Foundation Trust
city: London
country: United Kingdom
facility: National Hospital for Neurology and Neurosurgery
city: London
country: United Kingdom
facility: Great Ormond Street Hospital NHS Foundation Trust
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust
city: Salford
country: United Kingdom
facility: Salford Royal NHS Foundation Trust
Study officials:
affiliation: BioMarin Pharmaceutical
name: Medical Director, MD
role: STUDY_DIRECTOR
References: