In short
Clinical trials of Imiglucerase are studying its use in people with Gaucher disease, mainly type 1 and type 3. These studies look at safety, effectiveness, and brain-related outcomes in adult and pediatric patients. The trial data also compare Imiglucerase use in combination treatment settings.
Key points
- Clinical trials of Imiglucerase are focused on Gaucher disease, especially type 1 and type 3. The available trial data include both adult and pediatric patients. The studies are in Phase 2 and Phase 3 and look at safety, treatment effects, and biomarkers. Some trials also measure balance, coordination, and thinking skills in people with Gaucher disease type 3. The trial data show that Imiglucerase is being studied in combination treatment settings and in pediatric patients as part of broader Gaucher disease research.
Clinical trial overview
The trial data provided here show three interventional studies that include Imiglucerase in Gaucher disease research. The studies focus on Gaucher disease type 1 and type 3, with participants ranging from children to adults. Two trials are Phase 3 studies, and one is a Phase 2 study.
Phase 2 study in adult Gaucher disease type 3
NCT02843035 is a Phase 2 interventional trial in 18 participants with Gaucher disease type 3 and Gaucher disease type 1. The title shows that the study examined venglustat in combination with Cerezyme in adult patients with Gaucher disease type 3, and it also included a venglustat monotherapy extension phase. The study was completed.
This trial had several parts. Part 1 was a biomarker screening phase, which means the researchers looked for body signals that could help separate adult Gaucher disease type 3 from adult Gaucher disease type 1 and identify adults who could move into later treatment parts. Parts 2 and 3 studied short-term and long-term safety and tolerability, which means how well the treatment was handled by the body over time. Part 4 studied extended treatment with monotherapy, meaning treatment with only one study drug, in adults who stayed systemically stable on combination treatment.
The main outcomes were the number of participants with treatment emergent adverse events, which are new medical problems that appear after treatment starts, and changes in cerebrospinal fluid biomarkers, including lyso-glucosylceramide and glucosylceramide. These biomarkers are measurable substances that can help researchers understand disease activity in the brain and spinal fluid.
Phase 3 study in adult and pediatric Gaucher disease type 3
NCT05222906 is a Phase 3 interventional study with 50 participants with Gaucher disease type 3. The trial is authorised and includes both adults and pediatric patients aged 12 to under 18 years. The study compares venglustat with Cerezyme and evaluates efficacy and safety in this population.
The trial measures change in the Scale for the Assessment and Rating of Ataxia, often called SARA, and change in the Repeatable Battery for the Assessment of Neuropsychological Status, often called RBANS. SARA is used to measure ataxia, which is trouble with balance and coordination. RBANS is used to measure cognition, which means thinking and memory skills.
The trial data also show several study drugs and placebos in the intervention list, including Cerezyme, venglustat, and matching placebo tablets or placebo for Cerezyme. The source data do not give more detail on the treatment schedule, but they do show that this is a comparative trial designed to assess outcomes in a larger group than the Phase 2 study.
Phase 3 pediatric study in Gaucher disease type 1 and type 3
Trial 2024-510751-34-00 is a Phase 3 interventional study in 69 pediatric patients with Gaucher disease type 1 and type 3. The study was completed. Its title says it studied the safety and efficacy of eliglustat with or without Imiglucerase in pediatric patients with Gaucher disease type 1 and type 3.
The main outcomes were pharmacokinetic measures and adverse events. Pharmacokinetics, often shortened to PK, means how a drug moves through the body, including how much reaches the blood. The specific PK measures listed were Cmax, the highest concentration in plasma, and AUC, the area under the concentration-time curve, which shows total exposure over time. The trial also counted the number of adverse events in pediatric patients.
What the trials measure
Across these studies, the main focus is on safety, efficacy, and disease-related measurements. Safety is tracked through adverse events and treatment emergent adverse events. Efficacy means how well the treatment works, and it is measured in different ways depending on the study, such as SARA, RBANS, and biomarker changes.
One study measures biomarkers in cerebrospinal fluid, which is the fluid around the brain and spinal cord. Another study measures brain-related function with balance and thinking tests. The pediatric study measures how the treatment behaves in the body through PK values like Cmax and AUC.
Who the trials include
The trial populations are clearly different across the studies. One trial focuses on adults with Gaucher disease type 3 and also includes adults with type 1 in the biomarker screening part. Another trial includes adults and teenagers aged 12 to under 18 years with Gaucher disease type 3. The third trial includes children and teenagers aged 2 to under 18 years with Gaucher disease type 1 and type 3.
This mix of age groups shows that Imiglucerase-related trial research is being studied not only in adults, but also in pediatric patients. The source data do not provide full inclusion and exclusion rules, so the article can only describe the populations listed in the trial records.
Overall trial picture
In the provided trial data, Imiglucerase appears in studies that are part of broader Gaucher disease research, especially for type 3 disease. The studies range from early biomarker work in a small Phase 2 trial to larger Phase 3 studies in adults and children. The common theme is that researchers are trying to learn how treatment affects safety, brain-related symptoms, and measurable disease signals over time.