Study on Ivosidenib and Venetoclax With or Without Azacitidine for Treating Blood Cancers with IDH1 Mutation

Sponsor: M.D. Anderson Cancer Center

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT03471260

Provider View

This clinical trial is exploring the safety and effectiveness of combining the drugs ivosidenib and venetoclax, with or without azacitidine, to treat patients with blood cancers that have a specific IDH1 mutation. The study aims to find the best dose and see how well these drugs work together to stop cancer growth.

Patient Parameters

Program Overview

This study is testing a combination of drugs—ivosidenib and venetoclax, with or without azacitidine—to treat blood cancers with an IDH1 mutation. These drugs may help stop cancer cells from growing by blocking certain enzymes. Azacitidine, a chemotherapy drug, works by killing cancer cells or stopping them from dividing. The study aims to find the safest and most effective dose and to see if adding azacitidine improves treatment outcomes.

Description

The main goals of this study are to determine the safety and best dose of ivosidenib and venetoclax, with or without azacitidine, for patients with advanced blood cancers that have an IDH1 mutation. Researchers will also look at how well the treatment works in shrinking tumors and improving patient outcomes.

In the first phase, the study will focus on finding the safest dose of the drug combination. In the second phase, the study will evaluate how effective the treatment is in achieving a response, such as complete or partial remission, in patients with acute myeloid leukemia (AML).

The study will also explore how the drugs are processed in the body and identify any markers that might predict how well the treatment works or if there is resistance to it. Researchers will monitor the duration of response, survival rates, and any remaining cancer cells after treatment.

Participants will take venetoclax by mouth daily for the first 14 days of each cycle. Ivosidenib will be taken daily starting from day 15 of the first cycle and throughout subsequent cycles. Azacitidine may be given by injection or infusion for the first 7 days of each cycle. Each cycle lasts 28 days, and treatment continues as long as it is effective and side effects are manageable.

After completing the treatment, patients will have follow-up visits 30 days later and then monthly for up to three years to monitor their health and any long-term effects.

Eligibility Criteria

Inclusion Criteria

  • Must be over 18 years old.
  • Have a performance status score of less than 2, indicating good overall health.
  • Have a specific IDH1 mutation in their cancer, confirmed by a local lab.
  • Have relapsed or refractory acute myeloid leukemia (AML) or be newly diagnosed with AML and not eligible for standard chemotherapy. Patients with high-risk myelodysplastic syndromes (MDS) or myeloproliferative neoplasms (MPN) that have not responded to standard treatment may also be eligible.
  • Have adequate liver and kidney function based on specific blood tests.
  • Be willing and able to provide informed consent.
  • Have not received any treatment for at least 7 days before starting the study, unless the disease is rapidly progressing.
  • Male participants must agree to use effective contraception and avoid donating sperm during the study and for 90 days after the last dose.

Exclusion Criteria

  • Have a known allergy to ivosidenib or venetoclax.
  • Have previously been treated with ivosidenib or venetoclax.
  • Have any uncontrolled medical condition, infection, or psychiatric illness that could increase the risk of treatment.
  • Are taking other cancer treatments, except for specific exceptions like intrathecal chemotherapy for brain involvement or hydroxyurea for rapidly growing disease.
  • Are taking certain medications that affect how the study drugs work.
  • Have active graft-versus-host disease after a stem cell transplant.
  • Have severe digestive or metabolic issues that affect drug absorption.
  • Have another active cancer being treated.
  • Have a prolonged QT interval on an ECG, unless approved by the study doctor.
  • Have active hepatitis B, hepatitis C, or HIV infection.
  • Have a high white blood cell count, unless controlled with hydroxyurea.
  • Are pregnant, breastfeeding, or not using effective contraception if of childbearing potential.

Get This Program In Your Inbox

Receive detailed information about this treatment opportunity to review with your healthcare provider.