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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

Tracking Information
First Submitted Date November 6, 2008
First Posted Date November 10, 2008
Last Update Posted Date December 8, 2014
Study Start Date October 2008
Actual Primary Completion Date July 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: November 6, 2008)
  • Endurance [ Time Frame: Study Visit ]
  • Respiratory Function [ Time Frame: Study Visit ]
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT00787995 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Official Title A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Case-Only
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Subjects diagnosed with MPS IVA
Condition
  • MPS IV A
  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
Intervention Not Provided
Study Groups/Cohorts MPS IVA
Subjects with mucopolysaccharidosis IVA (Morquio Syndrome)
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Terminated
Actual Enrollment
 (submitted: December 5, 2014)
353
Original Estimated Enrollment
 (submitted: November 6, 2008)
300
Actual Study Completion Date July 2014
Actual Primary Completion Date July 2014   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Argentina,   Brazil,   Canada,   France,   Germany,   Italy,   Netherlands,   Taiwan,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00787995
Other Study ID Numbers MOR-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party BioMarin Pharmaceutical
Study Sponsor BioMarin Pharmaceutical
Collaborators Not Provided
Investigators
Study Director: Celeste Decker, MD BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date December 2014