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

This study has been terminated.
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT00787995
First received: November 6, 2008
Last updated: December 5, 2014
Last verified: December 2014
  Purpose

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
MPS IV A
Mucopolysaccharidosis IVA
Morquio A Syndrome

Study Type: Observational
Study Design: Observational Model: Case-Only
Official Title: A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Resource links provided by NLM:


Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • Endurance [ Time Frame: Study Visit ] [ Designated as safety issue: No ]
  • Respiratory Function [ Time Frame: Study Visit ] [ Designated as safety issue: No ]

Enrollment: 353
Study Start Date: October 2008
Study Completion Date: July 2014
Primary Completion Date: July 2014 (Final data collection date for primary outcome measure)
Groups/Cohorts
MPS IVA
Subjects with mucopolysaccharidosis IVA (Morquio Syndrome)

  Eligibility

Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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
  More Information

Additional Information:
No publications provided by BioMarin Pharmaceutical

Additional 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
Study First Received: November 6, 2008
Last Updated: December 5, 2014
Health Authority: United States: Institutional Review Board
United Kingdom: Research Ethics Committee
France: Institutional Ethical Committee
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

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

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

ClinicalTrials.gov processed this record on September 03, 2015