Efficacy and safety of enzyme replacement therapy for MPS I with 100 I.U./Kg recombinant human a-L-iduronidase (ALDURAZYME™).
Completed
- Conditions
- Mucopolysaccharidose type I.
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 35
Inclusion Criteria
1. The patient must give written informed consent;
2. If the patients is younger than 12 years, informed consent from his/her parents or his/her legal representative is necessary;
Exclusion Criteria
1. Patient is unable or unwilling to comply with the study protocol;
2. Parent(s) or legal representatives are unable or unwilling to comply with the evaluation program;
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 1. Improvement of joint mobility;<br /><br>2. Improvement of quality of life.
- Secondary Outcome Measures
Name Time Method 1. Improvement of sleep apnea registration;<br /><br>2. Improvement in 6-minute walk test;<br /><br>3. Improvement of cardiac geometry and function;<br /><br>4. Improvement in lung function;<br /><br>5. Improved motor performance (handicap status);<br /><br>6. Evaluation of visual acuity / performance;<br /><br>7. Evaluation of mental condition and social performance;<br /><br>8. Decrease of liver and/or spleen size as measured by ultrasound;<br /><br>9. Effect of dose and infusion rate on plasma enzyme levels and enzyme availability.