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

Morquio A Syndrome Clinical Trial

— MARS  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02294877
• Other study ID #: 110-504
• Status: Completed
• Start date: September 2014
• Est. completion date: February 21, 2024

Study information

• Verified date: March 2024
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical 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 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 418
• Est. completion date: February 21, 2024
• Est. primary completion date: February 21, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Morquio A Syndrome
• MPS IVA
• Mucopolysaccharidoses
• Mucopolysaccharidosis IV
• Mucopolysaccharidosis IV Type A

Intervention

Drug:
• Vimizim® (elosulfase alfa)
Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS)

Locations

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

Sponsors (2)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical	ICON plc

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	10 Years
Primary	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.	10 years
Secondary	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.	10 years