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Glasdegib

Glasdegib is an investigational drug being studied in clinical trials for the treatment of various blood cancers, including acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and chronic graft-versus-host disease (cGVHD). These trials aim to evaluate the safety and effectiveness of glasdegib when used alone or in combination with other treatments. The studies focus on different patient populations and treatment approaches, providing valuable insights into the potential benefits of this drug for patients with challenging blood disorders.

Table of Contents

What is Glasdegib?

Glasdegib is a medication used in the treatment of certain types of blood cancers. It is also known by its brand name DAURISMO™ and its research code PF-04449913[1][10]. Glasdegib is part of a class of drugs called Hedgehog pathway inhibitors, which work by blocking a specific cellular signaling pathway involved in cancer growth[4].

How Glasdegib Works

Glasdegib works by inhibiting a protein called Smoothened (SMO), which is part of the Hedgehog signaling pathway. This pathway is important in embryonic development but can be abnormally activated in some cancers. By blocking this pathway, glasdegib can help slow or stop the growth of cancer cells[4].

Conditions Treated with Glasdegib

Glasdegib is primarily used to treat:

  • Acute Myeloid Leukemia (AML): A type of blood cancer that affects the bone marrow and blood[1]
  • Myelodysplastic Syndrome (MDS): A group of disorders where the bone marrow doesn’t produce enough healthy blood cells[10]
  • Chronic Myelomonocytic Leukemia (CMML): A type of cancer that affects blood-forming cells in the bone marrow[10]
  • Chronic Graft-Versus-Host Disease (cGVHD): A complication that can occur after a stem cell or bone marrow transplant[9]

Glasdegib is often used in combination with other treatments, particularly for patients who are newly diagnosed with AML and are not candidates for intensive chemotherapy due to age or other health factors[1].

Clinical Trials and Research

Several clinical trials have been conducted or are ongoing to evaluate the effectiveness and safety of glasdegib:

  • A study (NCT03416179) is evaluating glasdegib in combination with intensive chemotherapy or azacitidine for previously untreated AML patients[1]
  • Another trial (NCT04111497) is investigating glasdegib for treating sclerosis associated with chronic graft-versus-host disease[9]
  • A study (NCT04231851) is examining the combination of CPX-351 (a chemotherapy drug) and glasdegib for newly diagnosed AML with MDS-related changes or therapy-related AML[10]

How Glasdegib is Administered

Glasdegib is typically taken orally as a tablet. The dosage and schedule can vary depending on the specific condition being treated and whether it’s being used alone or in combination with other medications. Common dosages include:

  • 100 mg taken once daily[1]
  • 50 mg, 75 mg, or 100 mg taken daily, depending on the patient’s needs and tolerability[10]

Treatment duration can range from several months to up to 2 years, depending on the patient’s response and the specific treatment protocol[1][9].

Side Effects and Safety

Like all medications, glasdegib can cause side effects. Common side effects may include:

  • Fatigue
  • Nausea
  • Decreased appetite
  • Muscle spasms
  • Changes in taste
  • Hair loss

More serious side effects can include:

  • Low blood cell counts (anemia, neutropenia, thrombocytopenia)
  • Abnormal heart rhythm (QT prolongation)
  • Liver problems

Patients are closely monitored during treatment for these and other potential side effects[1][9].

Drug Interactions

Glasdegib can interact with other medications. Of particular note:

  • Strong CYP3A4 inducers (like rifampin) can significantly reduce the effectiveness of glasdegib[8]
  • Medications that can prolong the QT interval should be used with caution in combination with glasdegib[6]

Always inform your healthcare provider about all medications, supplements, and herbal products you are taking.

Use in Special Populations

Research has been conducted to understand how glasdegib affects patients with certain health conditions:

  • Hepatic Impairment: A study (NCT03627754) evaluated the effect of moderate to severe liver impairment on glasdegib’s pharmacokinetics[3]
  • Renal Impairment: Another study (NCT03596567) investigated how kidney function affects the processing of glasdegib in the body[5]

These studies help determine if dose adjustments are necessary for patients with liver or kidney problems.

Aspect Details
Drug Name Glasdegib (PF-04449913)
Conditions Studied Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), Chronic Graft-Versus-Host Disease (cGVHD)
Administration Oral tablet, typically once daily
Dosage Range 25-200 mg daily, depending on the study
Combination Therapies Azacitidine, Cytarabine, Daunorubicin
Primary Outcomes Overall Survival, Safety Profile, Response Rates
Secondary Outcomes Event-Free Survival, Quality of Life Measures, Pharmacokinetics
Common Side Effects Fatigue, Nausea, Muscle Pain, Changes in Blood Cell Counts
Study Phases Phase 1, Phase 2, Phase 1b/2a
Patient Populations Newly Diagnosed AML, Refractory cGVHD, Various Age Groups

FAQ

  • What is glasdegib and how does it work? Glasdegib is an oral medication that inhibits a signaling pathway called the Hedgehog pathway. This pathway is involved in cancer cell growth and survival. By blocking this pathway, glasdegib may help slow down or stop the growth of cancer cells in certain blood disorders.
  • What types of blood cancers is glasdegib being studied for? Glasdegib is being investigated for several blood cancers, including acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and chronic graft-versus-host disease (cGVHD). It is being studied both as a single agent and in combination with other treatments.
  • How is glasdegib administered in clinical trials? In most trials, glasdegib is given as an oral tablet, typically taken once daily. The dosage and duration of treatment may vary depending on the specific trial and the condition being studied. Some trials test different doses to determine the most effective and safe amount.
  • What are some potential side effects of glasdegib? Common side effects observed in clinical trials include fatigue, nausea, muscle pain, and changes in blood cell counts. More serious side effects may include liver problems and changes in heart rhythm. However, the full range of side effects is still being studied, and patients in clinical trials are closely monitored for any adverse reactions.
  • How can I find out if I'm eligible for a glasdegib clinical trial? Eligibility for glasdegib clinical trials depends on various factors, including your specific diagnosis, previous treatments, and overall health. You can discuss clinical trial options with your healthcare provider or search for ongoing trials on websites like clinicaltrials.gov. Each trial has specific inclusion and exclusion criteria that determine eligibility.

Ongoing Clinical Trials on Glasdegib

  • Study on Advanced Soft-Tissue Sarcoma: Testing Futibatinib and Drug Combination for Patients with Unresectable or Metastatic Conditions

    Not recruiting

    1 1 1 1
    Investigated drugs:
    France

Glossary

  • Acute Myeloid Leukemia (AML): A type of blood cancer that starts in the bone marrow and quickly moves into the blood. It affects the production of normal blood cells and can progress rapidly if not treated.
  • Myelodysplastic Syndrome (MDS): A group of disorders where the bone marrow doesn't produce enough healthy blood cells. It can sometimes progress to acute myeloid leukemia.
  • Chronic Graft-Versus-Host Disease (cGVHD): A complication that can occur after a stem cell or bone marrow transplant, where the donor's immune cells attack the recipient's tissues.
  • Hedgehog Pathway: A signaling pathway in cells that plays a role in cell growth and development. In some cancers, this pathway can become overactive, contributing to tumor growth.
  • Complete Remission (CR): A treatment response where there are no detectable cancer cells in the body and blood cell counts have returned to normal levels.
  • Minimal Residual Disease (MRD): The small number of cancer cells that may remain in the body after treatment, even when a person is in remission.
  • Overall Survival (OS): The length of time from the start of treatment or diagnosis that patients are still alive.
  • Event-Free Survival (EFS): The length of time after treatment that a patient remains free of certain complications or events that the treatment was intended to prevent or delay.
  • Adverse Event (AE): Any unfavorable and unintended sign, symptom, or disease temporarily associated with the use of a medical treatment or procedure.
  • Pharmacokinetics: The study of how a drug is absorbed, distributed, metabolized, and eliminated by the body.

References

  1. https://clinicaltrials.gov/study/NCT03416179
  2. https://clinicaltrials.gov/study/NCT04842604
  3. https://clinicaltrials.gov/study/NCT03627754
  4. https://clinicaltrials.gov/study/NCT03162900
  5. https://clinicaltrials.gov/study/NCT03596567
  6. https://clinicaltrials.gov/study/NCT04111497
  7. https://clinicaltrials.gov/study/NCT03415867
  8. https://clinicaltrials.gov/study/NCT03270878
  9. https://clinicaltrials.gov/study/NCT02430545
  10. https://clinicaltrials.gov/study/NCT04231851