AG-120 for Blood Cancers
Trial Summary
Do I have to stop taking my current medications for the AG-120 trial?
The trial protocol does not specify if you need to stop taking your current medications, but it does exclude patients taking medications that prolong the QT interval (a measure of heart rhythm). It's best to discuss your specific medications with the trial team.
What data supports the effectiveness of the drug AG-120 (Ivosidenib) for blood cancers?
Ivosidenib has been shown to be effective in treating acute myeloid leukemia (AML) with IDH1 mutations, achieving complete remission in about 33% of patients with relapsed or refractory AML. It also improved survival rates when combined with azacitidine in newly diagnosed AML patients with IDH1 mutations.12345
Is ivosidenib (AG-120, Tibsovo) safe for humans?
Ivosidenib has been studied in patients with certain blood cancers and is generally considered safe, but it can cause serious side effects like differentiation syndrome (a condition where cancer cells rapidly change), high white blood cell counts, and heart rhythm changes. Common side effects include tiredness, nausea, diarrhea, and rash. Long-term safety is still being evaluated.12345
How is the drug ivosidenib unique for treating blood cancers?
Ivosidenib is unique because it specifically targets and inhibits the mutant IDH1 protein, which is responsible for producing a harmful substance called 2-hydroxyglutarate (2-HG) that contributes to cancer growth. This drug is taken orally and is particularly effective for patients with blood cancers that have an IDH1 mutation, offering a targeted approach compared to traditional chemotherapy.12346
What is the purpose of this trial?
The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced hematologic malignancies that harbor an IDH1 mutation. The first portion of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of AG-120 to determine maximum tolerated dose (MTD) and/or the recommended Phase II dose. The second portion of the study is a dose expansion phase where four cohorts of patients will receive AG-120 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose. Additionally, the study includes a substudy evaluating the safety and tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of AG-120 in subjects with relapsed or refractory myelodysplastic syndrome with an IDH1 mutation. Anticipated time on study treatment is until disease progression or unacceptable toxicity occurs.
Eligibility Criteria
Adults (18+) with advanced blood cancers like AML or MDS that have a specific IDH1 mutation can join. They must be able to undergo regular bone marrow, blood, and urine tests, not be pregnant, and have an ECOG performance status of 0-2. Adequate liver and kidney function is required. Those with severe heart problems, recent cancer treatments within 14 days (except hydroxyurea), active infections including HIV or hepatitis B/C, CNS leukemia symptoms or history of it without clinical suspicion are excluded.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Dose Escalation
Cohorts of patients receive ascending oral doses of AG-120 to determine maximum tolerated dose (MTD) and/or the recommended Phase II dose
Dose Expansion
Four cohorts of patients receive AG-120 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- AG-120
AG-120 is already approved in United States for the following indications:
- Acute myeloid leukemia (AML) with a susceptible IDH1 mutation
Find a Clinic Near You
Who Is Running the Clinical Trial?
Institut de Recherches Internationales Servier
Lead Sponsor