Phase 3
N=45
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
Mucopolysaccharidosis I · Hurler's Syndrome · Hurler-Scheie Syndrome · Scheie Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00146770 ↗Enrolled (actual)
45
Serious AEs
55.6%
Results posted
Jun 2009
Primary outcome: Primary: Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) — 51.0; 48.4; 47.7; 47.2 percent predicted FVC
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Aldurazyme (Biological); placebo (Biological)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Genzyme, a Sanofi Company
- Primary completion
- Mar 2005
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) |
51.0; 48.4; 47.7; 47.2; -3.3; -1.2 | — |
| PRIMARY Change From Baseline to Week 182 in Six Minute Walk Test (6MWT) |
348.3; 319.0; 367.7; 358.3; 19.4; 39.2 | — |
| SECONDARY Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI) |
14.6; 18.0; 9.8; 14.0; -4.8; -4.0 | — |
| SECONDARY Change From Baseline to Week 182 in Liver Volume |
1351.6; 1228.5; 1079.9; 1001.4; -17.6; -16.1 | — |
| SECONDARY Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score |
1.75; 1.99; 1.49; 1.56; -0.26; -0.43 | — |
| SECONDARY Change From Baseline to Week 182 in Active Joint Range of Motion (ROM) |
85.2; 96.1; 103.5; 109.2; 18.3; 13.1 | — |
Summary
This study is being conducted to collect additional long-term efficacy and safety data of Aldurazyme® (laronidase) patients with MPS I disease. Patients who were previously enrolled in the Phase 3 Double-Blind Study will be enrolled in this study.
Eligibility Criteria
Inclusion Criteria
- The patient or patient's legal guardian must provide written informed consent prior to any protocol-related procedures being performed.
- The patient must have successfully completed Study ALID-003-99 (who received 21 of 26 consecutive weekly infusions).
- The patient has not experienced any safety issues that would contraindicate participation in the Extension study.
- A female patient of childbearing potential must have a negative pregnancy test at entry
Exclusion Criteria
- The patient is pregnant or lactating.
- The patient has received an investigational drug within 30 days prior to the study enrollment.
- The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly interfere with study compliance including all prescribed evaluations and follow-up activities
Data sourced from ClinicalTrials.gov (NCT00146770). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.