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

BioMarin Pharmaceutical

Status: Terminated

Conditions: MPS IV A; Mucopolysaccharidosis IVA; Morquio A Syndrome

Study type: Observational

Funder types: Industry

Identifiers

NCT00787995

MOR-001

Details and patient eligibility

About

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.

Enrollment: 353 patients

Sex: All

Volunteers: No 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.

Trial design

353 participants in 1 patient group

MPS IVA

Description:

Subjects with mucopolysaccharidosis IVA (Morquio Syndrome)