Ivosidenib for Acute Myeloid Leukemia

Recruiting in Palo Alto (17 mi)

+1 other location

Overseen ByAmir Fathi, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Massachusetts General Hospital

Prior Safety Data

Breakthrough Therapy

Approved in 2 jurisdictions

Trial Summary

What is the purpose of this trial?This is a Phase 2 study of the study drug, ivosidenib (a mutant IDH1 inhibitor), compared to placebo, given to patients with IDH1-mutant acute myeloid leukemia (AML) after hematopoietic stem cell transplantation (HCT).

Eligibility Criteria

This trial is for adults aged 18-75 with IDH1-mutant acute myeloid leukemia who are undergoing or have undergone a bone marrow transplant. They must be in good health, with specific blood counts and organ functions within normal ranges, and agree to use contraception. Those with recent relapse/refractory disease, other cancers (unless cancer-free for 5 years), previous transplants, hepatitis B/C, heart failure/arrhythmias, or uncontrolled illnesses cannot join.

Inclusion Criteria

My leukemia is IDH1(R132) mutant type.

I am between 18 and 75 years old.

I can take care of myself but might not be able to do heavy physical work.

My blood counts and liver, kidney, and heart functions are within normal ranges.

I am scheduled for a stem cell transplant from a donor for my cancer.

I am of childbearing age and have a negative pregnancy test.

Exclusion Criteria

I have a history of serious heart rhythm problems.

I have had a stem cell transplant from a donor.

I have an active hepatitis B or C infection.

I have severe heart failure, dangerous irregular heartbeats, or poor heart pump function.

Participant Groups

The study compares ivosidenib—a drug targeting mutant IDH1 enzymes—to a placebo in patients after stem cell transplantation. It's designed to see if ivosidenib can help maintain remission from AML post-transplant by inhibiting the abnormal enzyme produced by mutated genes in these leukemia cells.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: IvosidenibExperimental Treatment1 Intervention

After screening and standard of care hematopoietic stem cell transplantation (HCT) and any standard treatment before and after the HSCT, Ivosidenib will be given orally (PO) once daily (QD) in 28-day continuous cycles. Ivosidenib is administered at a pre-determined dose with a goal of 500mg daily. Participants begin study treatment 45 to 90 days after HCT, and treatment will continue for up to 24 months. Study visits and assessments occur throughout study treatment and in follow-up. After the 24-month treatment period, participants are followed for up to 12 additional months.

Group II: PlaceboPlacebo Group1 Intervention

After screening and standard of care hematopoietic stem cell transplantation (HCT) and any standard treatment before and after the HSCT, Placebo will be given orally (PO) once daily (QD) in 28-day continuous cycles. Participants begin study treatment (placebo) 45 to 90 days after HCT, and treatment (placebo) will continue for up to 24 months. Study visits and assessments occur throughout study treatment period and in follow-up. After the 24-month treatment period, participants are followed for up to 12 additional months.