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

Sponsor

BioMarin Pharmaceutical (Industry)

Overall Status

Terminated

CT.gov ID

NCT00787995

Collaborator

(none)

353

Enrollment

Locations

Duration (Months)

23.5

Patients Per Site

0.3

Patients Per Site Per Month

Study Details

Study Description

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	Intervention/Treatment	Phase
MPS IV A Mucopolysaccharidosis IVA Morquio A Syndrome

Study Design

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 :

Oct 1, 2008

Actual Primary Completion Date :

Jul 1, 2014

Actual Study Completion Date :

Jul 1, 2014

Arms and Interventions

Arm	Intervention/Treatment
MPS IVA Subjects with mucopolysaccharidosis IVA (Morquio Syndrome)

Outcome Measures

Primary Outcome Measures

Endurance [Study Visit]
Respiratory Function [Study Visit]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

	City	State	Country
1	Oakland	California	United States
2	Chicago	Illinois	United States
3	Cordoba		Argentina
4	Porto Alegre		Brazil
5	Sao Paulo		Brazil
6	Montreal	Quebec	Canada
7	Lyon		France
8	Paris		France
9	Mainz		Germany
10	Monza		Italy
11	Amsterdam		Netherlands
12	Taipei		Taiwan
13	Birmingham		United Kingdom
14	London		United Kingdom
15	Manchester		United Kingdom