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

Tracking Information

First Received Date ^ICMJE

November 6, 2008

Last Updated Date

July 29, 2009

Start Date ^ICMJE

October 2008

Estimated Primary Completion Date

December 2009 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Endurance [ Time Frame: Study Visit ] [ Designated as safety issue: No ]
Respiratory Function [ Time Frame: Study Visit ] [ Designated as safety issue: No ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT00787995 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Original Secondary Outcome Measures ^ICMJE

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

A Multicenter, Multinational, Cross-sectional Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Brief Summary

This multicenter, multinational, cross-sectional study in subjects diagnosed with MPS IVA will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease.

Detailed Description

Study Phase

Study Type ^ICMJE

Observational

Study Design ^ICMJE

Case-Only, Cross-Sectional

Condition ^ICMJE

MPS IV A

Intervention ^ICMJE

Study Arms / Comparison Groups

Subjects with mucopolysaccharidosis IVA (Morquio Syndrome)

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

300

Completion Date

Estimated Primary Completion Date

December 2009 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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.
Diagnosis of MPS IVA, demonstrated by documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay.
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.

Gender

Both

Ages

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Candice F Henkel	US 1-866-961-8212
Contact: Candice F Henkel	Int'l +1-415-506-3580

Location Countries ^ICMJE

United States, France, United Kingdom

Administrative Information

NCT ID ^ICMJE

NCT00787995

Responsible Party

BioMarin Pharmaceutical Inc., BioMarin Pharmaceutical Inc.

Study ID Numbers ^ICMJE

MOR-001

Study Sponsor ^ICMJE

BioMarin Pharmaceutical

Collaborators ^ICMJE

Investigators ^ICMJE

Study Director:

Celeste Decker, MD

BioMarin Pharmaceutical

Information Provided By

BioMarin Pharmaceutical

Verification Date

July 2009

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP