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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03485677 | Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 | PHASE3 | COMPLETED | 57 | — | — | Apr 11, 2018 | Dec 12, 2025 | Jan 14, 2026 | 21 | Argentina, Canada +8 |
Maximum concentration (Cmax) of eliglustat in plasma
Area under the plasma eliglustat concentration-time curve (AUC)
Number of adverse events in pediatric patients
| Arm | Type | Description |
|---|---|---|
| Cohort 1: Eliglustat monotherapy | EXPERIMENTAL | Eliglustat for at least two years. Cohort 1 patients that experience significant clinical decline will receive rescue treatment. Rescue Treatment Step 1: Switch from eliglustat to imiglucerase monotherapy. Rescue Treatment Step 2: Patients who after 6 months of rescue therapy with imiglucerase monotherapy do not show improvement in the parameter(s) that led to the switch from eliglustat to imiglucerase, will then receive combination therapy with eliglustat + imiglucerase. |
| Cohort 2: Eliglustat plus imiglucerase | EXPERIMENTAL | Eliglustat plus imiglucerase for three years, at the dose of enzyme replacement therapy received before enrollment. After Week 52, Cohort 2 patients will switch to eliglustat monotherapy for the remainder of the study if the desired clinical response has been achieved. |
| Name | Type | Description |
|---|---|---|
| Eliglustat GZ385660 | DRUG | Pharmaceutical form: Capsule, Liquid Route of administration: Oral |
| Imiglucerase GZ437843 | DRUG | Pharmaceutical form: Powder for solution for infusion Route of administration: Intravenous |
Inclusion criteria : * The patient is 2 to \<18 years old at the time of informed consent. * Male and female patients with a clinical diagnosis of Gaucher disease (GD) type 1 or type 3 with documented deficiency of acid beta-glucosidase activity by enzyme assay and glucocerebrosidase (GBA) genotype...