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Eliglustat Tartrate

Eliglustat tartrate, also known as Genz-112638, is an oral medication being studied in clinical trials for the treatment of Gaucher disease, a genetic disorder that affects the body’s ability to break down certain fats. These trials aim to evaluate the efficacy, safety, and long-term effects of eliglustat tartrate in patients with different types of Gaucher disease, including those who have previously been treated with enzyme replacement therapy.

Table of Contents

What is Eliglustat Tartrate?

Eliglustat Tartrate, also known by its research name Genz-112638, is an oral medication developed for the treatment of Gaucher disease[1]. It is a small molecule drug that has been designed to be taken by mouth, offering a convenient alternative to enzyme replacement therapies that require intravenous infusions[4].

How Does Eliglustat Tartrate Work?

Eliglustat Tartrate works by regulating the Gaucher disease process in a unique way. Instead of replacing the missing enzyme (as in enzyme replacement therapy), it decreases the production of a fatty substance called glucosylceramide[1]. In Gaucher disease, this substance accumulates in cells, causing various health problems. By reducing its production, Eliglustat Tartrate helps to prevent the buildup of this harmful substance in the body’s cells, particularly in organs like the liver, spleen, and bone marrow[4].

What Conditions Does Eliglustat Tartrate Treat?

Eliglustat Tartrate is primarily used to treat Gaucher disease Type 1, which is the most common form of this rare genetic disorder[5]. Gaucher disease Type 1 is characterized by the following symptoms:

  • Splenomegaly: An enlarged spleen
  • Hepatomegaly: An enlarged liver
  • Thrombocytopenia: Low platelet count, which can lead to easy bruising and bleeding
  • Anemia: Low red blood cell count, causing fatigue and weakness
  • Bone disease: Can cause pain, fractures, and other skeletal problems
  • Decreased quality of life

Recent research is also exploring the use of Eliglustat Tartrate in treating Gaucher disease Type 3, which is a more severe form that affects the central nervous system[3].

How is Eliglustat Tartrate Administered?

Eliglustat Tartrate is taken orally in capsule form. The dosage and frequency can vary depending on the individual patient and their response to the medication. Typically, the treatment follows this pattern:

  1. Initial dose: Usually starts with 50 mg taken twice daily
  2. Dose adjustment: After 2-4 weeks, the dose may be adjusted based on blood tests that measure the concentration of the drug in the patient’s system
  3. Maintenance dose: Can range from 50 mg to 150 mg twice daily, depending on the patient’s needs and how they metabolize the drug[4][5]

It’s important to note that the dosage is carefully monitored and adjusted by healthcare professionals to ensure the best possible treatment outcome for each patient.

How Effective is Eliglustat Tartrate?

Clinical trials have shown promising results for Eliglustat Tartrate in treating Gaucher disease Type 1. The medication has demonstrated effectiveness in:

  • Reducing spleen and liver volume
  • Increasing hemoglobin levels (improving anemia)
  • Increasing platelet counts
  • Improving bone health
  • Enhancing quality of life for patients[1][5]

Long-term studies have shown that many patients remain stable on Eliglustat Tartrate treatment for several years, indicating its potential as a long-term management option for Gaucher disease[4].

What Are the Potential Side Effects?

As with any medication, Eliglustat Tartrate can have side effects. However, in clinical trials, it has generally been well-tolerated by patients. Common side effects may include:

  • Headache
  • Nausea
  • Diarrhea
  • Fatigue

It’s important to discuss any side effects with your healthcare provider. They can help manage these effects and determine if any adjustments to your treatment plan are necessary[1].

Ongoing Research and Future Prospects

Research on Eliglustat Tartrate is ongoing, with several clinical trials exploring its use in different patient populations and treatment scenarios. Some areas of current research include:

  • Use in pediatric patients: A study is underway to evaluate the efficacy and safety of Eliglustat in children and adolescents (ages 12 to 18) with Gaucher disease Types 1 and 3[3]
  • Long-term effectiveness: Studies are continuing to assess the long-term benefits and safety of Eliglustat Tartrate over several years of use[4]
  • Comparison with other treatments: Research is comparing Eliglustat Tartrate to existing treatments like enzyme replacement therapy to determine its relative effectiveness and potential advantages[4]

These ongoing studies aim to provide more comprehensive data on the use of Eliglustat Tartrate, potentially expanding its applications and improving treatment outcomes for patients with Gaucher disease.

Aspect Details
Drug Name Eliglustat tartrate (Genz-112638)
Disease Target Gaucher disease (Type 1 and Type 3)
Mechanism of Action Inhibits production of glucosylceramide
Administration Oral capsules (50mg, 100mg, 150mg)
Dosing Regimen Twice daily (BID), with dose adjustments based on plasma concentrations
Primary Outcomes Changes in spleen volume, liver volume, hemoglobin levels, platelet counts
Secondary Outcomes Bone mineral density, quality of life measures, biomarker levels
Study Populations Adults and pediatric patients (12-18 years old)
Study Durations 52 weeks to 9 years (including long-term follow-up)
Comparators Placebo, Imiglucerase (enzyme replacement therapy)

FAQ

  • What is Gaucher disease? Gaucher disease is a genetic disorder that results in a deficiency of an enzyme called acid beta-glucosidase. This enzyme deficiency leads to the accumulation of a fatty substance called glucosylceramide in various organs, particularly the liver, spleen, and bone marrow. This accumulation can cause a range of symptoms, including enlarged liver and spleen, low blood cell counts, and bone problems.
  • How does eliglustat tartrate work? Eliglustat tartrate is an oral medication that works by inhibiting the production of glucosylceramide, the fatty substance that accumulates in Gaucher disease. By reducing the production of this substance, eliglustat tartrate aims to decrease its buildup in cells and tissues, potentially improving the symptoms of Gaucher disease.
  • What are the main goals of the clinical trials studying eliglustat tartrate? The main goals of these clinical trials are to evaluate the efficacy, safety, and long-term effects of eliglustat tartrate in patients with Gaucher disease. Researchers are looking at various outcomes, including changes in organ size (spleen and liver), blood cell counts (hemoglobin and platelets), bone health, and quality of life measures. They are also assessing the drug's performance compared to existing treatments and its effectiveness in maintaining stability in patients previously treated with enzyme replacement therapy.
  • Who can participate in these clinical trials? The eligibility criteria vary among the different trials, but generally, they include adult patients (over 16 or 18 years old) with Gaucher disease type 1 or type 3. Some trials focus on patients who have not received previous treatment, while others include patients who have been stabilized on enzyme replacement therapy. There is also a trial specifically for pediatric patients aged 12 to 18 years old.
  • What are the potential benefits and risks of participating in these trials? Potential benefits include access to a new oral treatment for Gaucher disease, which may be more convenient than intravenous enzyme replacement therapy. Participants also receive close medical monitoring throughout the trial. However, as with any clinical trial, there are potential risks, including unknown side effects of the medication. It's important to note that the safety and efficacy of eliglustat tartrate are still being studied, and participants should discuss all potential risks and benefits with their healthcare providers before enrolling in a trial.

Ongoing Clinical Trials on Eliglustat Tartrate

  • 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

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

Glossary

  • Gaucher disease: A genetic disorder characterized by the deficiency of an enzyme called acid beta-glucosidase, leading to the accumulation of a fatty substance called glucosylceramide in various organs, particularly the liver, spleen, and bone marrow.
  • Eliglustat tartrate: An oral medication being studied for the treatment of Gaucher disease. It works by inhibiting the production of glucosylceramide, the fatty substance that accumulates in Gaucher disease.
  • Enzyme replacement therapy (ERT): A treatment approach for Gaucher disease that involves regular intravenous infusions of a manufactured enzyme to replace the deficient enzyme in the body.
  • Glucosylceramide: A fatty substance that accumulates in the cells of patients with Gaucher disease due to the lack of the enzyme needed to break it down.
  • Splenomegaly: An enlargement of the spleen, which is a common symptom in Gaucher disease.
  • Hepatomegaly: An enlargement of the liver, which is another common symptom in Gaucher disease.
  • Thrombocytopenia: A condition characterized by abnormally low levels of platelets in the blood, which can lead to easy bruising and bleeding.
  • Anemia: A condition where there is a deficiency of red blood cells or hemoglobin in the blood, leading to fatigue and other symptoms.
  • Bone mineral density (BMD): A measurement of the amount of calcium and other minerals in bones, used to assess bone strength and health.
  • T-score: A measure used in bone density scans that compares a person's bone density to that of a healthy young adult of the same gender.
  • Z-score: A measure used in bone density scans that compares a person's bone density to the average for their age, gender, and size.
  • Multiples of normal (MN): A unit of measurement used to express organ volumes (such as liver or spleen) in relation to what is considered normal for a person's body size.
  • Pharmacokinetics (PK): The study of how a drug is absorbed, distributed, metabolized, and eliminated by the body over time.
  • Quality of life (QoL): A measure of an individual's overall well-being and ability to function in daily life, often assessed in clinical trials to determine the impact of a treatment on patients' lives.

References

  1. https://clinicaltrials.gov/study/NCT00358150
  2. https://clinicaltrials.gov/study/NCT01074944
  3. https://clinicaltrials.gov/study/NCT06523517
  4. https://clinicaltrials.gov/study/NCT00943111
  5. https://clinicaltrials.gov/study/NCT00891202