A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
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| ClinicalTrials.gov Identifier: NCT00787995 |
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Recruitment Status :
Terminated
First Posted : November 10, 2008
Last Update Posted : December 8, 2014
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Sponsor:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
BioMarin Pharmaceutical
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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 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
| Group/Cohort |
|---|
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MPS IVA
Subjects with mucopolysaccharidosis IVA (Morquio Syndrome)
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Primary Outcome Measures :
- Endurance [ Time Frame: Study Visit ]
- Respiratory Function [ Time Frame: Study Visit ]
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.
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
| 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
| Study Director: | Celeste Decker, MD | BioMarin Pharmaceutical |
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| 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:
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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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Syndrome Mucopolysaccharidoses Mucopolysaccharidosis IV Osteochondrodysplasias 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 |