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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
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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

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


Outcome Measures
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Primary Outcome Measures :
  1. Endurance [ Time Frame: Study Visit ]
  2. Respiratory Function [ Time Frame: Study Visit ]

Eligibility Criteria
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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
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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
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Additional Information:

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

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Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT00787995    
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