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Label indication ALDURAZYME ® is indicated for the treatment of: adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and patients with the Scheie form of MPS I who have moderate to severe symptoms. FDA label
Also known as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), Laronidase, Recombinant human alpha L iduronidase
Aldurazyme · 4 trials · 5 indications
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.
Immune tolerance induction success was defined as development of an anti-laronidase immunoglobulin G (IgG) antibody titer less than or equal to (\<=) 1:3200 after 24 weeks of receiving full-dose (0.58 mg/kg) laronidase therapy.
| 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. |
| Cohort 1 | EXPERIMENTAL | Tolerance Induction Period (TIP): Cyclosporine A (CsA) starting at 5 milligram per kilogram (mg/kg) orally three times daily until the target trough concentration of at least 350 nanogram per milliliter (ng/mL) (preferably 400 ng/mL) achieved along with azathioprine (Aza) 2.5 mg/kg/day orally. Once target CsA trough level achieved and maintained for at least 1 week, participants received laronidase 0.058 mg/kg (low-dose) once weekly intravenous (IV) infusion (starting from Day 1) up to Week 12. CsA and Aza were gradually discontinued. Immune Challenge Period (ICP): following TIP, laronidase dose increased to 0.12 mg/kg once weekly IV infusion for 1 week followed by 0.25 mg/kg once weekly IV infusion for 1 week and then 0.58 mg/kg (full-dose) once weekly IV infusion up to Week 39. |
| Cohort 2 | EXPERIMENTAL | TIP: CsA starting at 6.7 mg/kg orally three times daily until the target trough concentration of at least 350 ng/mL (preferably 400 ng/mL) achieved along with Aza 5 mg/kg orally every other day. Once target CsA trough level achieved and maintained for at least 1 week, participants received laronidase 0.058 mg/kg (low-dose) once weekly IV infusion (starting from Day 1) up to Week 18. CsA and Aza were gradually discontinued. ICP: following TIP, laronidase dose increased to 0.12 mg/kg once weekly IV infusion for 1 week followed by 0.25 mg/kg once weekly IV infusion for 1 week and then 0.58 mg/kg (full-dose) once weekly IV infusion up to Week 45. |
| 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 |
| Laronidase | BIOLOGICAL | 0.058 mg/kg - 0.58 mg/kg IV infusion weekly. |
| Cyclosporine A (CsA) | DRUG | Orally three times daily. |
| Azathioprine (Aza) | DRUG | Orally either every day for Cohort 1 or every other day for Cohort 2. |
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...
Aldurazyme is used to treat Mucopolysaccharidosis I (MPS I), a rare inherited lysosomal storage disorder that includes Hurler, Hurler-Scheie, and Scheie syndromes. It is a replacement therapy studied in patients across these MPS I disease subtypes, including children under five years old.
Aldurazyme is developed by Sanofi, which trades under the ticker SNY. The company is the sponsor behind the clinical development program for this MPS I therapy.
Aldurazyme has been studied in Phase 3 clinical development for Mucopolysaccharidosis I, with completed Phase 3 trials and one completed Phase 2 trial. All three of its registered trials are completed, and none are currently active.
Aldurazyme has been evaluated in three completed trials: NCT00258011, a Phase 3 study in Japan; NCT00146770, a Phase 3 extension study across the United States, Brazil, Canada, Germany, Italy, the Netherlands, and the United Kingdom; and NCT00146757, a Phase 2 pharmacokinetics study in patients under five.
Yes. Aldurazyme is also known as laronidase, or recombinant human alpha-L-iduronidase. The names Aldurazyme and laronidase refer to the same MPS I therapy, and trial titles use both names interchangeably.
The three completed Aldurazyme trials enrolled a combined total of 68 patients. NCT00146770 was the largest with 45 participants, followed by NCT00146757 with 20 and NCT00258011 with 3.