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

A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome). - A Cross-sectional Clinical Assessment of Subjects with MPS IV A.

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON36408

Enrollment

Registered

2010-03-03

Start date

2009-07-01

Completion date

Unknown

Last updated

2024-05-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

metabolic disease syndrome of Morquio

Interventions

None listed

Eligibility

Age

2 Years to 99 Years

Inclusion criteria

Inclusion criteria: • Willing and able to provide written, signed informed consent, or in the case of subjects age

Exclusion criteria

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.

Design outcomes

Secondary

Measure	Time frame
To characterize the spectrum of symptoms and biochemical abnormalities.	—

Primary

Measure	Time frame
To quantify the endurance and respiratory function.	—

Countries

Netherlands

Outcome results