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

Morquio A Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00787995
• Other study ID #: MOR-001
• Status: Terminated
• Phase: N/A
• First received: November 6, 2008
• Last updated: December 5, 2014
• Start date: October 2008
• Est. completion date: July 2014

Study information

• Verified date: December 2014
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review BoardUnited Kingdom: Research Ethics CommitteeFrance: Institutional Ethical CommitteeFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 353
• Est. completion date: July 2014
• Est. primary completion date: July 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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.

Study Design

Observational Model: Case-Only

Related Conditions & MeSH terms

• Morquio A Syndrome
• MPS IV A
• Mucopolysaccharidoses
• Mucopolysaccharidosis IV
• Mucopolysaccharidosis IVA
• Syndrome

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  Argentina,  Brazil,  Canada,  France,  Germany,  Italy,  Netherlands,  Taiwan,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Endurance		Study Visit	No
Primary	Respiratory Function		Study Visit	No

External Links

•   BioMarin Pharmaceutical Inc. website