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Imiglucerase

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.

Table of Contents

Clinical trial overview

The trial data provided here show three interventional studies that include Imiglucerase in Gaucher disease research.[1][2][3] The studies focus on Gaucher disease type 1 and type 3, with participants ranging from children to adults.[1][2][3] Two trials are Phase 3 studies, and one is a Phase 2 study.[1][2][3]

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.[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.[1] The study was completed.[1]

This trial had several parts.[1] 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.[1] 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.[1] Part 4 studied extended treatment with monotherapy, meaning treatment with only one study drug, in adults who stayed systemically stable on combination treatment.[1]

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.[1] These biomarkers are measurable substances that can help researchers understand disease activity in the brain and spinal fluid.[1]

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.[2] The trial is authorised and includes both adults and pediatric patients aged 12 to under 18 years.[2] The study compares venglustat with Cerezyme and evaluates efficacy and safety in this population.[2]

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.[2] SARA is used to measure ataxia, which is trouble with balance and coordination.[2] RBANS is used to measure cognition, which means thinking and memory skills.[2]

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.[2] 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.[2]

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.[3] The study was completed.[3] 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.[3]

The main outcomes were pharmacokinetic measures and adverse events.[3] Pharmacokinetics, often shortened to PK, means how a drug moves through the body, including how much reaches the blood.[3] 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.[3] The trial also counted the number of adverse events in pediatric patients.[3]

What the trials measure

Across these studies, the main focus is on safety, efficacy, and disease-related measurements.[1][2][3] Safety is tracked through adverse events and treatment emergent adverse events.[1][3] 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.[1][2]

One study measures biomarkers in cerebrospinal fluid, which is the fluid around the brain and spinal cord.[1] Another study measures brain-related function with balance and thinking tests.[2] The pediatric study measures how the treatment behaves in the body through PK values like Cmax and AUC.[3]

Who the trials include

The trial populations are clearly different across the studies.[1][2][3] One trial focuses on adults with Gaucher disease type 3 and also includes adults with type 1 in the biomarker screening part.[1] Another trial includes adults and teenagers aged 12 to under 18 years with Gaucher disease type 3.[2] The third trial includes children and teenagers aged 2 to under 18 years with Gaucher disease type 1 and type 3.[3]

This mix of age groups shows that Imiglucerase-related trial research is being studied not only in adults, but also in pediatric patients.[1][2][3] The source data do not provide full inclusion and exclusion rules, so the article can only describe the populations listed in the trial records.[1][2][3]

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.[1][2] The studies range from early biomarker work in a small Phase 2 trial to larger Phase 3 studies in adults and children.[1][2][3] The common theme is that researchers are trying to learn how treatment affects safety, brain-related symptoms, and measurable disease signals over time.[1][2][3]

Trial ID Phase Condition studied Status Enrollment
NCT02843035 Phase 2 Gaucher disease type 3, Gaucher disease type 1 Completed 18
NCT05222906 Phase 3 Gaucher’s disease type III Authorised 50
2024-510751-34-00 Phase 3 Gaucher’s disease type I, Gaucher’s disease type III Completed 69

FAQ

  • What are the Imiglucerase clinical trials studying? The trials are studying safety, effectiveness, and biomarker changes in people with Gaucher disease. Some studies also look at brain-related symptoms such as ataxia, which means trouble with balance and coordination.
  • Who can take part in these trials? The trial data include adult patients and pediatric patients, including children and teenagers. The studies focus on people with Gaucher disease type 1 and type 3.
  • What phases are the Imiglucerase trials? The trial data include Phase 2 and Phase 3 studies. Phase 2 studies usually focus on early testing of safety and biological effects, while Phase 3 studies compare treatment effects in larger groups.
  • What conditions are being studied? The trials study Gaucher disease type 1 and Gaucher disease type 3. One trial focuses on adult patients with type 3, while another includes both adult and pediatric patients with type 3.
  • What outcomes are measured in these trials? The studies measure adverse events, which are unwanted medical problems during the trial, and changes in biomarkers, which are body signals that can show disease activity. Some trials also measure balance and thinking skills using special rating scales.

Ongoing Clinical Trials on Imiglucerase

  • Study on the Effectiveness and Safety of Venglustat for Adults and Children with Gaucher Disease Type 3

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy

  • Study of Eliglustat alone or with Imiglucerase in children and adolescents aged 2-17 years with Gaucher disease type 1 and type 3

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    France Italy Spain

  • Study of Venglustat and Imiglucerase for Adult Patients with Gaucher Disease Type 3

    Not recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    Germany

Glossary

  • Gaucher disease: A genetic disease studied in these trials. It can affect the body in different ways depending on the type, including type 1 and type 3.
  • Gaucher disease type 1: A form of Gaucher disease included in one pediatric trial. The trial data do not give more detail about symptoms for this type.
  • Gaucher disease type 3: A form of Gaucher disease studied in the adult and pediatric trials. The studies focus on this type because it can involve brain-related symptoms.
  • Adult patients: People who are grown up. One trial studies adults with Gaucher disease type 3.
  • Pediatric patients: Children and teenagers. One trial includes patients from 2 to under 18 years old.
  • Interventional study: A clinical trial where participants receive a treatment or comparison treatment so researchers can measure results.
  • Phase 2: An earlier trial phase that often looks at safety and early signs of benefit in a smaller group.
  • Phase 3: A later trial phase that usually studies treatment effects in a larger group and compares options.
  • Adverse events: Medical problems that happen during a study. They may or may not be caused by the treatment.
  • Biomarker: A measurable body signal, such as a substance in fluid, that can help show how a disease is behaving.
  • Cerebrospinal fluid: The fluid around the brain and spinal cord. One trial measures biomarkers in this fluid.
  • Ataxia: Trouble with balance and coordination. One trial measures this with a rating scale.

References