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Ivosidenib

Ivosidenib, also known as AG-120 or Tibsovo, is an innovative drug being studied in various clinical trials for its potential in treating different types of cancer, particularly those with IDH1 mutations. These trials aim to evaluate the drug’s effectiveness, safety, and impact on patients’ quality of life across different cancer types and treatment scenarios.

Table of Contents

What is Ivosidenib?

Ivosidenib, also known by its brand name TIBSOVO, is a medication used to treat certain types of cancer[1]. It’s also referred to as AG-120, AG120, or S95031 in some medical literature[2]. This drug is part of a class of medications called IDH1 inhibitors, which target a specific genetic mutation found in some cancer cells.

How Does Ivosidenib Work?

Ivosidenib works by blocking the activity of a mutated form of the IDH1 enzyme (isocitrate dehydrogenase 1). This enzyme, when mutated, can lead to the overproduction of substances that contribute to the growth of cancer cells. By inhibiting this mutated enzyme, ivosidenib may help slow down or stop the growth of cancer cells[1].

What Conditions Does Ivosidenib Treat?

Ivosidenib is primarily used to treat several types of cancer that have a specific IDH1 mutation. These include:

  • Acute Myeloid Leukemia (AML): A type of blood and bone marrow cancer[3]
  • Cholangiocarcinoma: A type of bile duct cancer[4]
  • Solid Tumors: Various types of cancers that form solid masses[1]
  • Myeloproliferative Neoplasms (MPNs): A group of blood cancers where the bone marrow makes too many blood cells[5]

It’s important to note that ivosidenib is specifically used for cancers that have an IDH1 mutation. Your doctor will need to perform genetic testing on your cancer cells to determine if this treatment is appropriate for you.

How is Ivosidenib Administered?

Ivosidenib is taken orally, usually once daily. The typical dose is 500 mg, but your doctor may adjust this based on your specific condition and how you respond to the treatment[4]. It’s important to take ivosidenib exactly as prescribed by your healthcare provider.

Some key points about taking ivosidenib:

  • It can be taken with or without food
  • You should avoid eating grapefruit or drinking grapefruit juice while taking this medication
  • It’s best to take it at approximately the same time each day[3]

Current Clinical Trials

Ivosidenib is currently being studied in several clinical trials to explore its effectiveness in different scenarios:

  • As a maintenance therapy after stem cell transplantation for AML patients[2]
  • In combination with other drugs like azacitidine for newly diagnosed AML patients[6]
  • For treating advanced solid tumors in children and adolescents[1]
  • In combination with ruxolitinib for advanced myeloproliferative neoplasms[5]

Potential Side Effects

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

  • Fatigue
  • Nausea
  • Changes in blood cell counts
  • Electrolyte imbalances

More serious side effects can occur, such as a condition called differentiation syndrome, which can cause fever, difficulty breathing, and other symptoms. It’s crucial to report any new or worsening symptoms to your healthcare provider immediately[6].

Special Considerations

There are some important factors to consider when taking ivosidenib:

  • Drug Interactions: Ivosidenib may interact with other medications. Be sure to inform your doctor about all medications, supplements, and herbal products you’re taking[6].
  • Liver Function: Your doctor may need to monitor your liver function while you’re taking this medication[7].
  • Pregnancy and Breastfeeding: If you’re pregnant, planning to become pregnant, or breastfeeding, discuss the potential risks and benefits with your healthcare provider.
  • QT Prolongation: Ivosidenib may affect your heart rhythm. Your doctor may perform regular electrocardiograms (ECGs) to monitor this[6].

Remember, ivosidenib is a powerful medication that should only be used under the close supervision of a healthcare professional. Always follow your doctor’s instructions and report any concerns or side effects promptly.

Aspect Details
Drug Name Ivosidenib (AG-120, Tibsovo)
Mechanism Inhibitor of mutant IDH1 protein
Cancer Types Studied AML, cholangiocarcinoma, solid tumors, lymphomas, histiocytic disorders with IDH1 mutations
Administration Oral, typically once daily
Key Outcomes Measured Progression-free survival, overall survival, objective response rate, safety, quality of life
Trial Phases Phase 1 and Phase 2 studies
Special Populations Pediatric patients, Japanese patients, patients with hepatic impairment
Combination Therapies Studied with azacitidine in some trials
Post-Transplant Use Investigated as maintenance therapy after stem cell transplantation

FAQ

  • What is Ivosidenib and how does it work? Ivosidenib is a drug that targets and inhibits mutated IDH1 proteins. It works by blocking the growth of cancer cells that have specific genetic changes in the IDH pathway, potentially slowing down or stopping the progression of certain cancers.
  • What types of cancers are being studied with Ivosidenib? Clinical trials are exploring Ivosidenib's effectiveness in various cancers, including acute myeloid leukemia (AML), cholangiocarcinoma (bile duct cancer), solid tumors, lymphomas, and histiocytic disorders with IDH1 mutations.
  • How is Ivosidenib administered in clinical trials? In most trials, Ivosidenib is given as an oral medication, typically taken once daily. The dosage and duration of treatment may vary depending on the specific trial and condition being studied.
  • What are some potential side effects of Ivosidenib? While side effects can vary, clinical trials are closely monitoring for adverse events. Common areas of focus include changes in blood counts, liver function, and potential interactions with other medications. Specific side effects are being evaluated in each trial.
  • How long do Ivosidenib clinical trials typically last? The duration of Ivosidenib trials can vary widely, from several months to several years. Some studies involve treatment periods of up to 24 months, with additional follow-up periods to assess long-term outcomes and survival rates.

Ongoing Clinical Trials on Ivosidenib

  • Study on the Safety of Ivosidenib and Azacitidine for Adults with Newly Diagnosed IDH1m Acute Myeloid Leukemia Not Eligible for Intensive Chemotherapy

    Recruiting

    1 1 1 1
    Investigated drugs:
    Austria Belgium France Greece Hungary Ireland +5

  • Study on Ivosidenib for Patients with Previously Treated Advanced or Metastatic Cholangiocarcinoma

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium France Germany Ireland Italy +4

  • A study to evaluate the safety and efficacy of ivosidenib, decitabine, and cedazuridine in adults with newly diagnosed acute myeloid leukemia who are older than 60 or cannot receive intensive chemotherapy.

    Not yet recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Spain

  • A Study of Ivosidenib and Azacitidine for Patients Over 55 with Acute Myeloid Leukemia and IDH1 Mutation in Complete Remission

    Not yet recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    France

  • Study on Molidustat and Ivosidenib for Adults with Relapsed or Refractory IDH1-Mutated Acute Myeloid Leukemia or Myelodysplastic Syndrome

    Not yet recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Germany

  • Study of Ivosidenib for Patients with Previously Treated Advanced or Metastatic Cholangiocarcinoma

    Not yet recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Germany The Netherlands Sweden

  • Study on the Safety and Effectiveness of Ivosidenib or Enasidenib with Azacitidine for Patients with Newly Diagnosed Acute Myeloid Leukemia with IDH1 or IDH2 Mutations

    Not recruiting

    1 1 1
    Investigated drugs:
    France Germany Italy The Netherlands

  • Study on the Effectiveness of Ivosidenib in Patients with IDH1 Mutated Myelodysplastic Syndrome

    Not recruiting

    1 1 1
    Investigated drugs:
    France

  • Study of Ivosidenib or Enasidenib with Chemotherapy for Patients with Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with IDH1 or IDH2 Mutation

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Estonia Finland France Germany +6

  • Study of Ivosidenib and Azacitidine for Patients Aged 18 and Older with Untreated Acute Myeloid Leukemia with IDH1 Mutation

    Not recruiting

    1 1 1
    Investigated drugs:
    France Germany Italy Poland Spain

Glossary

  • IDH1: Isocitrate dehydrogenase 1, an enzyme that, when mutated, can contribute to the growth of certain cancers.
  • Acute Myeloid Leukemia (AML): A type of cancer that affects the blood and bone marrow, characterized by rapid growth of abnormal white blood cells.
  • Cholangiocarcinoma: A type of cancer that forms in the bile ducts, which are tubes that connect the liver to the small intestine.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Pharmacodynamics (PD): The study of how a drug affects the body, including its mechanism of action and relationship between drug concentration and effect.
  • Progression-Free Survival (PFS): The length of time during and after treatment that a patient lives with the disease without it getting worse.
  • Overall Survival (OS): The length of time from either the date of diagnosis or the start of treatment that patients are still alive.
  • Objective Response Rate (ORR): The proportion of patients whose cancer shrinks or disappears after treatment.
  • Graft-versus-Host Disease (GVHD): A potential complication following a stem cell or bone marrow transplant where the donated cells attack the recipient's body.
  • Measurable Residual Disease (MRD): The small number of cancer cells that may remain in the body during or after treatment, often undetectable by standard tests.

References

  1. https://clinicaltrials.gov/study/NCT04195555
  2. https://clinicaltrials.gov/study/NCT06707493
  3. https://clinicaltrials.gov/study/NCT05030441
  4. https://clinicaltrials.gov/study/NCT06081829
  5. https://clinicaltrials.gov/study/NCT06291987
  6. https://clinicaltrials.gov/study/NCT06181734
  7. https://clinicaltrials.gov/study/NCT03282513