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

NCT02294877 | Completed

• 1 other identifier: 110-504
• Study Type: observational
• Target: 418
• Locations: 16 countries
• Sites: 72

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.

Conditions

Mucopolysaccharidosis IV Type A; Morquio A Syndrome; MPS IVA

Outcome Measures

Primary Outcomes (2)

• 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

• 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 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

  10 years

Interventions

Vimizim® (elosulfase alfa) DRUG

Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS)

Also known as: BMN 110

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• Patients currently participating in a BMN 110 (elosulfase alfa) clinical trial

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead
• ICON plc: collaborator

Study Sites (79)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

University of Southern California

Los Angeles, California, 90033, United States

Location

Children's Hospital of Orange County

Orange, California, 92868, United States

Location

A.I. Dupont Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Children's National Health System

Washington D.C., District of Columbia, 20010, United States

Location

Miami Children's Hospital

Miami, Florida, 33155, United States

Location

Emory University

Decatur, Georgia, 30033, United States

Location

Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96826, United States

Location

Ann & Robert H. Lurie Children's Hospital

Chicago, Illinois, 60611, United States

Location

University Of Iowa

Iowa City, Iowa, 52524, United States

Location

University of Louisville Research Foundation Inc. - Kosair Charities Pediatric Clinical Research Unit

Louisville, Kentucky, 40202, United States

Location

Children's Hospital of Boston

Boston, Massachusetts, 02115, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States

Location

Children's Specialty Center of Nevada

Las Vegas, Nevada, 89109, United States

Location

Atlantic Health System

Morristown, New Jersey, 07960, United States

Location

NYU-Langone Medical Center School of Medicine

New York, New York, 10016, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

UTHealth McGovern Medical School

Houston, Texas, 77030, United States

Location

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23507, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98145-5005, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Perth Children's Hospital

Perth, Nedlands, 6009, Australia

Location

Children's Hospital of Westmead

Westmead, New South Wales, 2145, Australia

Location

Westmead Hospital

Westmead, New South Wales, Australia

Location

Children's Health Queensland Hospital and Health Service

South Brisbane, Queensland, 4101, Australia

Location

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

Location

Royal Children's Hospital - Victoria

Parkville, Victoria, 3052, Australia

Location

Royal Perth Hospital

Perth, Western Australia, 6847, Australia

Location

Universitätsklinik Graz

Graz, 8036, Austria

Location

Medical University of Vienna

Vienna, 1090, Austria

Location

Antwerp University Hospital

Edegem, 2650, Belgium

Location

Adult Metabolic Diseases Clinic Vancouver General Hospital

Vancouver, British Columbia, V5Z 1M9, Canada

Location

Children's & Women's Centre of British Columbia

Vancouver, British Columbia, V6H 3N1, Canada

Location

Sainte-Justine Hospital

Montreal, Quebec, H3T 1C5, Canada

Location

McGill University Health Centre

Montreal, Quebec, H4A 3J1, Canada

Location

Université de Sherbrooke

Sherbrooke, Quebec, J1H 5N4, Canada

Location

Klinika Dětského a Dorostového Lékařstvi

Prague, 12000, Czechia

Location

Copenhagen University Hospital, Klinik For Sjaeldne Handicap

Copenhagen, Dk-2100, Denmark

Location

Hôpital Femme Mère Enfant

Bron, 69677, France

Location

Hospital Necker

Paris, 75743, France

Location

Hopital d'enfants - Hopitaux de Brabois

Vandœuvre-lès-Nancy, 54500, France

Location

Temple Street Children's University Hospital

Dublin, Ireland

Location

Azienda Policlinico Vittorio Emanuele, presidio G Rodolico, Università di Catania

Catania, 95125, Italy

Location

Azienda Ospedaliero Meyer

Florence, 50139, Italy

Location

Azienda Ospedaliero San Gerardo di Monza

Monza, 20090, Italy

Location

Federico II University Hospital

Naples, 80131, Italy

Location

Hospital Kuala Lumpur

Kuala Lumpur, 50586, Malaysia

Location

University of Malay Medical Center

Kuala Lumpur, Malaysia

Location

Academic Medical Center - University of Amsterdam

Amsterdam, 1105 AZ, Netherlands

Location

Rotterdam University Hospital Medical Centre

Rotterdam, 3015 GE, Netherlands

Location

The Children Memorial Health Institute

Warsaw, 04-730, Poland

Location

Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra)

Coimbra, 3000-062, Portugal

Location

Centro Hospitalar Lisboa Centro - Hospital de Dona Estefânia

Lisbon, 1169-045, Portugal

Location

Centro Hospitalar Lisboa Norte

Lisbon, Portugal

Location

San Jorge Children's Hospital, Centro de Investigaciones Clinicas

San Juan, Commonwealth, 00912, Puerto Rico

Location

Changhua Christian Hospital (CCH)

Changhua, Changhua County, 50006, Taiwan

Location

Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, San-Min District, 80756, Taiwan

Location

Chang Gung Memorial Hospital

Taoyuan, Taoyuan County, 33305, Taiwan

Location

Kaohsiung Veterans General Hospital

Kaohsiung City, 81362, Taiwan

Location

Chang Gung Memorial Hospital Kaohsiung Branch

Kaohsiung City, 83301, Taiwan

Location

China Medical University and Hospital

Taichung, 40402, Taiwan

Location

Taichung Veterans General Hospital

Taichung, 40705, Taiwan

Location

National Cheng Kung University (NCKU) Hospital

Tainan, 70403, Taiwan

Location

National Taiwan University Hospital

Taipei, 10050, Taiwan

Location

MacKay Memorial Hospital

Taipei, 10449, Taiwan

Location

Taipei Veterans General Hospital

Taipei, 11217, Taiwan

Location

Belfast City Hospital

Belfast, BT9 7AB, United Kingdom

Location

University Hospital Birmingham - Selly Oaks

Birmingham, B15 2WB, United Kingdom

Location

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

Royal Free NHS Foundation Trust

London, NW3 2QG, United Kingdom

Location

National Hospital for Neurology and Neurosurgery

London, WC1N 3BG, United Kingdom

Location

Great Ormond Street Hospital NHS Foundation Trust

London, WC1N 3JH, United Kingdom

Location

Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust

Manchester, M13 9WL, United Kingdom

Location

Salford Royal NHS Foundation Trust

Salford, M6 8HD, United Kingdom

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood serum; urine samples

MeSH Terms

Conditions

Mucopolysaccharidosis IV

Interventions

GALNS protein, human

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Director, MD

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 10 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 28, 2014
• First Posted: November 19, 2014
• Study Start: September 1, 2014
• Primary Completion: February 21, 2024
• Study Completion: February 21, 2024
• Last Updated: March 5, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

NL (2); IE (1); PT (3); IT (4); PL (1); PR (1); AU (2); MY (2); CA (5); TW (11); US (26); CZ (1); DK (1); FR (3); BE (1); GB (8)