atural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI. Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme.

• Conditions: Mucopolysaccharidosis type I, II and VI.; MedDRA version: 12.0Level: LLTClassification code 10028095Term: Mucopolysaccharidosis IV; MedDRA version: 12.0Level: LLTClassification code 10056886Term: Mucopolysaccharidosis I; MedDRA version: 12.0Level: PTClassification code 10056889Term: Mucopolysaccharidosis II

• Registration Number: EUCTR2007-001453-26-NL

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-11-13
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

1. The patient should have a biochemically confirmed deficiency of a-L-iduronidase (MPS I). iduronidate-2-sulfatase (MPS II), or N-acetylgalactosamine-4-sulfatase (MPS VI); or a confirmed mutation in the gene encoding for a-L-iduronidase (MPS I). iduronidate-2-sulfatase (MPS II, or N-acetylgalactosamine-4-sulfatase (MPS VI).
2. The patient has had least one evaluation through which the severity of the disease has been assessed and the urgency of enzyme therapy can be determined.
3. Written informed consent must be obtained from the patient and/or from the patient's parent/guardian if the patient is under 18 years of age.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. The patient (or parent/legal guardian) is unable or unwilling to comply with the study protocol.
2. The patient has severe neurological involvement as evidenced by:
* total or subtotal absence of cortical activity.
* untreatable seizures
* loss of (almost) all abilities to communicate.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified