Ivosidenib Access Program for Patients with Relapsed or Hard-to-Treat AML with an IDH1 Mutation
Sponsor: Agios Pharmaceuticals, Inc.
ClinicalTrials ID:NCT03245424
This program provides access to the drug ivosidenib for patients with acute myeloid leukemia (AML) that has returned or is not responding to treatment, specifically for those with an IDH1 mutation.
Patient Parameters
| Parameter | Options |
|---|
Program Overview
The purpose of this program is to offer the drug ivosidenib to patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific genetic mutation called IDH1. This program allows patients to receive the medication as a single treatment to help manage their condition.
Description
Patients who qualify for this program will take ivosidenib, a medication given by mouth once a day in 28-day cycles. Treatment will continue until the disease progresses, side effects become unacceptable, the patient or doctor decides to stop, the patient undergoes a stem cell transplant, the patient passes away, or the program ends. If a patient achieves complete remission, undergoes a stem cell transplant, and then relapses, they may rejoin the program after consulting with the Medical Monitor. The program will close if ivosidenib receives marketing approval. If the program ends for other reasons, access to the drug will be evaluated based on the reason for termination.
Patients will have initial assessments to confirm eligibility. After that, treatment and check-ups will follow the usual care standards at the study site, and records will be kept in the patient's chart. If the study drug is stopped, it is recommended that the patient returns for a safety check 28 days after the last dose.
Safety checks will be done regularly according to the care standards for patients taking ivosidenib. These checks include pregnancy tests, heart monitoring (ECG), lab tests, vital signs, physical exams, and monitoring for any significant side effects. The severity of any side effects will be graded using a standard system.
Eligibility Criteria
Inclusion Criteria
- Be 12 years or older and weigh at least 100 lbs.
- Have acute myeloid leukemia (AML) that has returned or is not responding to treatment.
- Have a confirmed IDH1 mutation in the cancer cells.
- Be able to understand and willing to sign a consent form. A legal representative can consent for those unable to do so.
- Have a performance status score of 0 to 3, indicating varying levels of activity.
- Have a platelet count of at least 20,000/µL (transfusions are allowed to reach this level).
- Have adequate liver function, with specific lab values within set limits.
- Have adequate kidney function, with a creatinine clearance over 30 mL/min.
- Be recovered from any significant side effects of previous cancer treatments.
- Women of childbearing potential must agree to pregnancy tests before starting the study drug and use effective contraception during the study and for 90 days after the last dose.
Exclusion Criteria
- Eligible for another clinical trial or have access to potentially curative treatment.
- Previously treated with an IDH1 inhibitor and did not respond.
- Had a stem cell transplant within 60 days before starting ivosidenib or have significant complications from a transplant.
- Received cancer treatment or investigational drugs within 14 days before starting ivosidenib.
- Taking certain medications that interact with ivosidenib unless they can be switched or monitored.
- Pregnant or breastfeeding.
- Have a severe infection or unexplained fever.
- Have a known allergy to ivosidenib.
- Have severe heart conditions or a prolonged QT interval on an ECG.
- Have active hepatitis B or C, or uncontrolled HIV.
- Have any other medical or psychological condition that may interfere with participation in the study.
- Have life-threatening complications of leukemia.
Get This Program In Your Inbox
Receive detailed information about this treatment opportunity to review with your healthcare provider.