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Givinostat

Givinostat is an experimental drug that is currently being studied in clinical trials for several different medical conditions. It belongs to a class of drugs called histone deacetylase (HDAC) inhibitors. Researchers are investigating givinostat’s potential to treat muscular dystrophy, blood disorders, arthritis, and other diseases. This article summarizes some of the key clinical trials evaluating givinostat’s safety and effectiveness.

Table of Contents

What is Givinostat?

Givinostat, also known as ITF2357, is a medication being studied for its potential to treat various medical conditions. It belongs to a class of drugs called histone deacetylase (HDAC) inhibitors[1]. HDAC inhibitors work by affecting how genes are expressed in cells, which can have wide-ranging effects on the body.

Conditions Treated with Givinostat

Research is ongoing to evaluate Givinostat’s effectiveness in treating several conditions:

  • Polycythemia Vera (PV): A blood disorder where the body produces too many red blood cells[1]
  • Duchenne Muscular Dystrophy (DMD): A genetic disorder characterized by progressive muscle degeneration and weakness[2]
  • Becker Muscular Dystrophy (BMD): A milder form of muscular dystrophy[3]
  • Chronic Myeloproliferative Neoplasms: A group of blood disorders where the bone marrow produces too many blood cells[4]
  • Juvenile Idiopathic Arthritis: A type of arthritis that affects children[5]

How Givinostat Works

Givinostat works by inhibiting histone deacetylases, which are enzymes that play a role in gene expression. By blocking these enzymes, Givinostat can affect how genes are turned on or off in cells. This mechanism is believed to have anti-inflammatory effects and may help slow down the progression of certain diseases[4].

In muscular dystrophy, for example, Givinostat is thought to counter the pathophysiological and degenerative mechanisms causing muscle insufficiency[6]. This could potentially help preserve muscle function and slow disease progression.

How Givinostat is Administered

Givinostat is typically administered orally (by mouth) in the form of:

  • Oral suspension: A liquid form of the medication, usually at a concentration of 10 mg/mL[2]
  • Capsules: Hard gelatin capsules containing 50 mg, 75 mg, or 100 mg of Givinostat[4]

The dosage and frequency of administration can vary depending on the condition being treated, the patient’s weight, and other factors. In many studies, Givinostat is taken twice daily with food[2][6].

Clinical Trials and Research

Givinostat is currently being studied in various clinical trials to assess its safety and effectiveness. Some key findings from these trials include:

  • In Polycythemia Vera, Givinostat combined with hydroxyurea has shown promise in controlling blood cell counts and reducing disease-related symptoms[1]
  • For Duchenne Muscular Dystrophy, studies have indicated that Givinostat may help improve muscle function and slow disease progression[7]
  • In Becker Muscular Dystrophy, research is ongoing to evaluate Givinostat’s effects on muscle histology (structure) and function[3]
  • For chronic myeloproliferative neoplasms, long-term studies are assessing Givinostat’s safety and efficacy[4]

Potential Side Effects

As with any medication, Givinostat may cause side effects. Common side effects observed in clinical trials include:

  • Gastrointestinal issues such as diarrhea, nausea, or vomiting[1]
  • Changes in blood cell counts, including a decrease in platelets (thrombocytopenia)[3]
  • Fatigue or tiredness[7]

It’s important to note that the safety profile of Givinostat is still being studied, and patients in clinical trials are closely monitored for any adverse effects[6].

Condition Trial Phase Key Findings
Duchenne muscular dystrophy Phase 2/3 Improved muscle histology and function in some measures; generally well-tolerated
Becker muscular dystrophy Phase 2 Reduced muscle fibrosis in biopsies; mixed results on functional outcomes
Polycythemia vera Phase 2 Reduced need for phlebotomies in some patients; manageable safety profile
Juvenile idiopathic arthritis Phase 2 Improved symptoms in subset of patients; further study needed

FAQ

  • What conditions is givinostat being studied to treat? Givinostat is being investigated as a potential treatment for several conditions, including Duchenne muscular dystrophy, Becker muscular dystrophy, polycythemia vera, and juvenile idiopathic arthritis. Clinical trials are evaluating its effects on muscle function, blood cell production, inflammation, and disease progression in these disorders.
  • How is givinostat administered? In clinical trials, givinostat is typically administered orally as a liquid suspension or capsules. The dosage and frequency varies depending on the specific trial and condition being studied, but it is often given twice daily with food.
  • What are some of the potential side effects of givinostat? Some potential side effects observed in clinical trials include diarrhea, nausea, decreased platelet counts, and changes in blood cell counts. However, the full safety profile is still being evaluated in ongoing studies.
  • How long do the clinical trials for givinostat typically last? The duration of givinostat clinical trials varies, but many last between 6-18 months for the main treatment period. Some trials also include longer-term extension phases to evaluate long-term safety and efficacy.
  • Is givinostat approved for use by regulatory agencies yet? Givinostat is still considered an investigational drug and is not yet approved for general use by regulatory agencies like the FDA. It can only be accessed through participation in clinical trials at this time while research is ongoing.

Ongoing Clinical Trials on Givinostat

  • Long-Term Safety and Efficacy Study of Givinostat for Patients with Duchenne Muscular Dystrophy Previously Treated with Givinostat

    Recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium France Germany Italy The Netherlands Spain

  • Study on the Safety and Effects of Givinostat in Young Children with Duchenne Muscular Dystrophy (Ages 2 to Less Than 6 Years)

    Recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Italy The Netherlands

  • Study Comparing Givinostat and Hydroxyurea for Patients with High-Risk Polycythemia Vera

    Recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Bulgaria Croatia France Germany Hungary +5

  • Efficacy and Safety of Givinostat for Non-Ambulant Patients with Duchenne Muscular Dystrophy

    Recruiting

    3 1
    Investigated diseases:
    Investigated drugs:
    Belgium Czechia France Germany Italy The Netherlands +3

  • Study on the Long-term Effects of Givinostat for Patients with JAK2V617F Positive Chronic Myeloproliferative Neoplasms

    Not recruiting

    2 1 1
    Investigated drugs:
    Italy

Glossary

  • Histone deacetylase (HDAC) inhibitor: A class of drugs that block enzymes involved in removing acetyl groups from histone proteins, which can affect gene expression. HDAC inhibitors like givinostat are being studied for various diseases.
  • Duchenne muscular dystrophy (DMD): A genetic disorder characterized by progressive muscle degeneration and weakness, typically affecting boys. It is caused by a mutation in the gene for dystrophin protein.
  • Becker muscular dystrophy (BMD): A milder form of muscular dystrophy related to DMD, also caused by mutations in the dystrophin gene but with some functional dystrophin produced.
  • Polycythemia vera: A blood disorder where the bone marrow produces too many red blood cells, leading to thickened blood and increased risk of blood clots.
  • Juvenile idiopathic arthritis: A type of arthritis that affects children, causing joint pain and inflammation. It includes several subtypes and can persist into adulthood.
  • Pharmacokinetics: The study of how a drug is absorbed, distributed, metabolized and eliminated by the body over time.
  • Maximum Tolerated Dose (MTD): The highest dose of a drug that does not cause unacceptable side effects, determined during early phase clinical trials.
  • Adverse event: Any unfavorable and unintended sign, symptom, or disease associated with the use of a medical treatment, whether or not considered related to the treatment.

References

  1. https://clinicaltrials.gov/study/NCT00928707
  2. https://clinicaltrials.gov/study/NCT03373968
  3. https://clinicaltrials.gov/study/NCT03238235
  4. https://clinicaltrials.eu/trial/study-on-the-long-term-effects-of-givinostat-for-patients-with-jak2v617f-positive-chronic-myeloproliferative-neoplasms/
  5. https://clinicaltrials.gov/study/NCT01261624
  6. https://clinicaltrials.eu/trial/study-on-givinostat-for-non-ambulant-patients-with-duchenne-muscular-dystrophy/
  7. https://clinicaltrials.gov/study/NCT01761292