A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

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ClinicalTrials.gov Identifier: NCT00787995

Recruitment Status : Terminated

First Posted : November 10, 2008

Last Update Posted : December 8, 2014

Sponsor:

Information provided by (Responsible Party):

BioMarin Pharmaceutical

Study Description

Brief Summary:

This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.

Condition or disease
MPS IV A Mucopolysaccharidosis IVA Morquio A Syndrome

Study Design

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Study Type :	Observational
Actual Enrollment :	353 participants
Observational Model:	Case-Only
Official Title:	A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Study Start Date :	October 2008
Actual Primary Completion Date :	July 2014
Actual Study Completion Date :	July 2014

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: Mucopolysaccharidosis type IV

Genetic and Rare Diseases Information Center resources: Mucopolysaccharidosis Mucopolysaccharidosis Type IV Mucopolysaccharidosis Type IVA

U.S. FDA Resources

Groups and Cohorts

Group/Cohort
MPS IVA Subjects with mucopolysaccharidosis IVA (Morquio Syndrome)

Outcome Measures

Primary Outcome Measures :

Endurance [ Time Frame: Study Visit ]
Respiratory Function [ Time Frame: Study Visit ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:	Child, Adult, Older Adult
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Subjects diagnosed with MPS IVA

Criteria

Inclusion Criteria:

Willing and able to provide written, signed informed consent, or in the case of subjects age < 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.
Willing and able to comply with all study procedures.

Exclusion Criteria:

Use of any investigational product or investigational medical device within 30 days prior to screening.
Previous hematopoietic stem cell transplant (HSCT).
Concurrent disease or condition that would interfere with study participation or pose a safety concern.

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00787995

Locations

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United States, California

Oakland, California, United States
United States, Illinois

Chicago, Illinois, United States
Argentina

Cordoba, Argentina
Brazil

Porto Alegre, Brazil

Sao Paulo, Brazil
Canada, Quebec

Montreal, Quebec, Canada
France

Lyon, France

Paris, France
Germany

Mainz, Germany
Italy

Monza, Italy
Netherlands

Amsterdam, Netherlands
Taiwan

Taipei, Taiwan
United Kingdom

Birmingham, United Kingdom

London, United Kingdom

Manchester, United Kingdom

Sponsors and Collaborators

BioMarin Pharmaceutical

Investigators

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Study Director:	Celeste Decker, MD	BioMarin Pharmaceutical

More Information

Additional Information:

BioMarin Pharmaceutical Inc. website This link exits the ClinicalTrials.gov site

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Martell LA, Cunico RL, Ohh J, Fulkerson W, Furneaux R, Foehr ED. Validation of an LC-MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome. Bioanalysis. 2011 Aug;3(16):1855-66. doi: 10.4155/bio.11.172.

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Responsible Party:	BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:	NCT00787995 History of Changes
Other Study ID Numbers:	MOR-001
First Posted:	November 10, 2008 Key Record Dates
Last Update Posted:	December 8, 2014
Last Verified:	December 2014

Keywords provided by BioMarin Pharmaceutical:


Morquio A Syndrome Mucopolysaccharidosis IV type A MPS IV Type A Mucopolysaccharidosis IVA MPS IVA Lysosomal Storage Disorder	LSD N-acetylgalactosamine-6-sulfatase galactose-6-sulfatase GALNS enzyme replacement therapy ERT

Additional relevant MeSH terms:

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Osteochondrodysplasias Mucopolysaccharidoses Mucopolysaccharidosis IV Syndrome Disease Pathologic Processes Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors	Genetic Diseases, Inborn Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases

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