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Laronidase · 1 trial · 1 indication
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 |
|---|---|---|
| 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 |
|---|---|---|
| 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 is required from the parent(s) or legal guardian(s) prior to any protocol-related procedures being performed. (A separate informed consent will be requested from the parent(s) for their genotyping, which is independent of the inclusion) * Participant's...
Laronidase is an investigational monoclonal antibody being studied for the treatment of Mucopolysaccharidosis I, a rare genetic disorder. It is currently in Phase 1 clinical development, with one completed trial involving 7 participants. The drug is not yet approved and remains under investigation for this condition.
Laronidase is a monoclonal antibody, but its specific molecular target has not been disclosed in available information. The drug is being evaluated for its potential role in treating Mucopolysaccharidosis I, though the exact mechanism of action has not been detailed in public clinical trial data.
Laronidase is developed by Sanofi, a global biopharmaceutical company listed on the stock exchange under the ticker SNY. Sanofi is conducting clinical research on this drug for the treatment of Mucopolysaccharidosis I, a rare disease indication.
Laronidase is in Phase 1 clinical development. One Phase 1 trial has been completed, with no active trials currently ongoing. The drug remains investigational and has not received regulatory approval for any indication.
Laronidase has one completed clinical trial registered under NCT00741338, titled 'Immune Tolerance Study With Aldurazyme® (Laronidase)'. This Phase 1 study enrolled 7 participants with Mucopolysaccharidosis I and was conducted in Brazil and Russia. The trial was controlled but not double-blinded.
Laronidase is also known as Aldurazyme, as indicated by the clinical trial title referencing Aldurazyme® (Laronidase). The drug is being studied under this alternative name, and the completed Phase 1 trial used this designation in its official title.