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

Status: Active, not recruiting
Location: See all (77) locations...
Intervention Type: Drug
Study Type: Observational [Patient Registry]
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.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: No
View:

• 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

Locations
United States
Arkansas
Arkansas Children's Hospital
Little Rock
Arizona
Phoenix Children's Hospital
Phoenix
California
Children's Hospital of Los Angeles
Los Angeles
University of Southern California
Los Angeles
Children's Hospital of Orange County
Orange
Washington, D.c.
Children's National Health System
Washington
Delaware
A.I. Dupont Hospital for Children
Wilmington
Florida
Miami Children's Hospital
Miami
Georgia
Emory University
Decatur
Hawaii
Kapiolani Medical Center for Women and Children
Honolulu
Iowa
University Of Iowa
Iowa City
Illinois
Ann & Robert H. Lurie Children's Hospital
Chicago
Kentucky
University of Louisville Research Foundation Inc. - Kosair Charities Pediatric Clinical Research Unit
Louisville
Massachusetts
Children's Hospital of Boston
Boston
Michigan
University of Michigan
Ann Arbor
Minnesota
Children's Hospitals and Clinics of Minnesota
Minneapolis
North Carolina
Duke University Medical Center
Durham
New Jersey
Atlantic Health System
Morristown
Nevada
Children's Specialty Center of Nevada
Las Vegas
New York
NYU-Langone Medical Center School of Medicine
New York
Pennsylvania
Children's Hospital of Philadelphia
Philadelphia
Children's Hospital of Pittsburgh of UPMC
Pittsburgh
Texas
UTHealth McGovern Medical School
Houston
Virginia
Children's Hospital of the King's Daughters
Norfolk
Washington
Seattle Children's Hospital
Seattle
Wisconsin
Children's Hospital of Wisconsin
Milwaukee
Other Locations
Australia
Royal Adelaide Hospital
Adelaide
Royal Children's Hospital - Victoria
Parkville
Perth Children's Hospital
Perth
Royal Perth Hospital
Perth
Children's Health Queensland Hospital and Health Service
South Brisbane
Children's Hospital of Westmead
Westmead
Westmead Hospital
Westmead
Austria
Universitätsklinik Graz
Graz
Medical University of Vienna
Vienna
Belgium
Antwerp University Hospital
Edegem
Canada
McGill University Health Centre
Montreal
Sainte-Justine Hospital
Montreal
Université de Sherbrooke
Sherbrooke
Adult Metabolic Diseases Clinic Vancouver General Hospital
Vancouver
Children's & Women's Centre of British Columbia
Vancouver
Denmark
Copenhagen University Hospital, Klinik For Sjaeldne Handicap
Copenhagen
France
Hôpital Femme Mère Enfant
Bron Cedex
Hospital Necker
Paris Cedex 15
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
Azienda Ospedaliero Meyer
Firenze
Azienda Ospedaliero San Gerardo di Monza
Monza
Federico II University Hospital
Naples
Malaysia
Hospital Kuala Lumpur
Kuala Lumpur
University of Malay Medical Center
Kuala Lumpur
Netherlands
Academic Medical Center - University of Amsterdam
Amsterdam
Rotterdam University Hospital Medical Centre
Rotterdam
Poland
The Children Memorial Health Institute
Warszawa
Portugal
Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra)
Coimbra
Centro Hospitalar Lisboa Norte
Lisboa
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
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung
Kaohsiung Veterans General Hospital
Kaohsiung
Chang Gung Memorial Hospital Kaohsiung Branch
Kaohsiung City
China Medical University and Hospital
Taichung
Taichung Veterans General Hospital
Taichung
National Cheng Kung University (NCKU) Hospital
Tainan
MacKay Memorial Hospital
Taipei
National Taiwan University Hospital
Taipei
Chang Gung Memorial Hospital
Taoyuan City
United Kingdom
Belfast City Hospital
Belfast
Birmingham Children's Hospital
Birmingham
University Hospital Birmingham - Selly Oaks
Birmingham
Great Ormond Street Hospital NHS Foundation Trust
London
National Hospital for Neurology and Neurosurgery
London
Royal Free NHS Foundation Trust
London
Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust
Manchester
Salford Royal NHS Foundation Trust
Salford
Time Frame
Start Date: September 2014
Estimated Completion Date: September 2024
Participants
Target number of participants: 427
Authors
Ngu Hock Lock, Merete Ljugberg, JoAnn Cheung, Huey Yin Leong, Viviana Brafa, Erin McAndrews, Serena Motto, Lauren Hitchens, Kaustuv Bharracharya, Danika Anganoo, Ana Van der Ploeg, Diogo Nuno Fonseca da Cruz, Jennifer Parrott, Ana Cristina Ferreira, Vikas Bhambani, Marissa Ferraris, Miriam Rigoldo, Alice Donati, Paula Garcia, Elena Procopio, Martin Magner, Phillipe Campeau, Karolina Stephien, Ayyanar Kanyalakshmi, Luisa Diogo, Michah Tong, Vassiliki Konstantopoulo, Lynne Ierardi-Curto, Stephanie Offord, William Wilcox, Michele Ford, Kara Goodin, Suma Shankar, Benjamin M Helm, Arlene Bayreder, Thomas Burrow, Raymond Wang, Heather Lau, Nadene Henderson, Angela Berg, Gerard Vockley, Areeg El-Gharbawy, Heidi Peters, Susan Hale, Donald Basel, Alexander Asamoah, Shanna Widera, Pranoot Tanpaiboon, Colleen Ditro, Maureen Cleary, Priya Kishnani, Damara Ortiz, Joel Charrow, Janice Fletcher, Rebecca Olson, Alan Ikeda, Samantha A Vergano, Shoji Yano, Klane White, Laurie Seaver, Erika Vucko, McKenzie Karelus, Elizabeth A Sellars, Barbara Burton, Nancy J. Mendelsohn, Mislen Bauer, Gordon Gowans, Adolfo Garnica, William Mackenzie, Leah Hecht, Amy Kritzer, Katherine G Langley, Angela Duker, Stefania Sala, John Mitchell, Sylvia Stockler-Ispiroglu, Juan A Santiago-Cornier, Emanuela Marrocco, Cinzia Galimberti, Annarita Pittala, Christina Lampe, Suresh Vijayaraghavan, Elisabeth Jameson, Nesrin Karabul, Reena Sharma, Charlotte Dawson, Gisela Wilcox, Saikat Santra, Atulkumar Mehta, Ana Jovanovic, Nicole Muschol, Thomas Lücke, Alexander Broomfield, Francois Feillet, Abra Roberts, Premala Muthukumarasamy, Kyla Almeida, Vera Anastasoaie, Hui Bein Chew, Silvia Sequeira, Angela Sun, Federica Raudino, Bruno Maranda, Roberto Della Casa, Ardeshir Ahmad Monavari, Sebastien Levesque, Sandra Sirrs, Fiona Stewart, Robert Wallerstein, Jiri Zeman, Dorothea Moeslinger, Lorne Clarke, Michaela Brunner-Krainz, Anita Inwood, Frits Wijburg, Derralynn Hughes, William Rhead, Michael Bober, Agata Fiumara, Simona Fecarotta, Ina Kerr, Brooke Blessing-Spangler, Carrie E Mishoe, Angela M Casey, A. Corina Moorehead, Josephine Hughes, Thong Meow-Keong, Elizabeth Neumeister-Chisholm, Nicola Longo, Nik Farhana Nik Abdul Rashid, Zahra Bakhtiar, Janice Sullivan, Ina Knerr, Simon Jones, Elaine Murphy, Ahmad Monavari, Ellen Crushell, Christian Hendriksz, Patricio Ricardo da Terra Aguiar, Shuan-Pei Lin, Olaf Bodamer, Uma Ramaswami, Serena Gasperini, Anna Tylki-Szymanska, Joanne Hughes, Richard Chang, Tarekegn G Hiwot, Francois Eyskens, Christine Dali, Rossella Parini, Nathalie Guffon, Robin Lachmann
Sponsors
Collaborators: ICON plc
Leads: BioMarin Pharmaceutical

This content was sourced from clinicaltrials.gov

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