Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00258011 | Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease | PHASE3 | COMPLETED | 3 | — | — | Dec 1, 2005 | Oct 1, 2006 | Mar 7, 2014 | 2 | Japan |
| NCT00146770 | Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients | PHASE3 | COMPLETED | 45 | — | — | May 1, 2001 | Mar 1, 2005 | Apr 3, 2015 | 25 | United States, Brazil +5 |
| NCT00146757 | A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old | PHASE2 | COMPLETED | 20 | — | — | Oct 1, 2002 | May 1, 2005 | Apr 3, 2015 | 4 | France, Germany +2 |
Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.
Percent Predicted Forced Vital Capacity: the maximal exhaled breath volume following a maximal inhaled breath. Overall change from Baseline to Week 182 in percent predicted FVC = (observed value)/(predicted value) \* 100%). A higher value indicates a greater response.
Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response.
AUC∞ is a measure of the total exposure to a drug.
Half-life is the time it takes for the concentration of drug in plasma to decline by 50%.
CL is volume of the body fluid cleared of the drug per unit of time.
Vz is the volume that relates the amount of drug in the body after absorption is complete to the concentration of drug in the plasma.
| Arm | Type | Description |
|---|---|---|
| Aldurazyme (laronidase) treatment | EXPERIMENTAL | Patients received weekly infusions of JC0498 (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight for up to 73 weeks. |
| Placebo/Aldurazyme | ACTIVE_COMPARATOR | Patients received placebo for 26 weeks in the Double-Blind Study then received 182 weeks of Aldurazyme (0.58 mg/kg every week) in this Extension Study; patients received a total of 182 weeks of Aldurazyme. |
| Aldurazyme/Aldurazyme | ACTIVE_COMPARATOR | Patients received 26 weeks of Aldurazyme in the Double-Blind Study and then received 182 weeks of Aldurazyme in this Extension Study; patients received a total of 208 weeks of Aldurazyme. |
| Aldurazyme (rhIDU) 100 U/kg ONLY every week | EXPERIMENTAL | Patients received Aldurazyme (recombinant human alpha-L-iduronidase (rhIDU)) once per week at a dose of 100 Units/kg (approximately 0.58 mg/kg) for up to 52 weeks - labeled dose. |
| Aldurazyme (rhIDU) 100-200 U/kg every week | EXPERIMENTAL | After receiving 100 Units/kg dose of Aldurazyme (rhIDU) for the first 25 weeks, patients enrolling after January 1, 2004 were eligible to receive an increased dose of 200 Units/kg from Week 26 onwards if the patient's urinary glycosaminoglycan (uGAG) levels were \>200µg/mg creatinine at Week 22. |
| Name | Type | Description |
|---|---|---|
| Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | BIOLOGICAL | 0.58 mg/kg every week |
| Aldurazyme | BIOLOGICAL | Placebo for 26 weeks then 0.58 mg/kg Aldurazyme every week for 182 weeks |
| placebo | BIOLOGICAL | Placebo for 26 weeks |
Inclusion Criteria: * Written informed consent/assent of the patient or written informed consent of the parent(s) or the legal guardian(s), depending on the age of the patient, is required prior to any protocol-related procedures being performed; this includes information regarding hematopoietic st...