Actively Recruiting
A Randomized, Placebo-Controlled Phase 2 Study of Ivosidenib as Maintenance Therapy for IDH1-Mutant Acute Myeloid Leukemia Following Stem Cell Transplantation
Led by Massachusetts General Hospital · Updated on 2026-04-28
75
Participants Needed
4
Research Sites
104 weeks
Total Duration
On this page
Sponsors
M
Massachusetts General Hospital
Lead Sponsor
S
Servier
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying whether ivosidenib, a drug that blocks a mutation called IDH1, can help prevent relapse in patients with acute myeloid leukemia (AML) who have this mutation and have received a stem cell transplant. This phase 2 trial compares ivosidenib to a placebo to see if it is beneficial after hematopoietic stem cell transplantation (HCT). The Food and Drug Administration (FDA) has approved ivosidenib for other uses but not yet for this specific post-transplant setting. Participants will start treatment between 45 and 90 days after their stem cell transplant. Those in the ivosidenib group will take the drug orally once daily in 28-day cycles for up to 24 months. The placebo group will follow the same schedule with a matching inactive tablet. After this treatment period, participants will be followed for an additional 12 months. Both groups receive standard care before, during, and after transplant. During the study, participants will have regular visits and assessments to monitor their health, including blood tests and checks for disease relapse. Researchers will measure how long participants remain relapse-free, overall survival, treatment side effects, and complications like graft versus host disease. The total study participation is about 3.5 years from screening through follow-up. The study is supported by a pharmaceutical company providing the drug and funding for research activities.
CONDITIONS
Brief Title
Ivosidenib as Post-HSCT Maintenance for AML
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 to 75 years
- Pathologically confirmed diagnosis of IDH1(R132)-mutant acute myeloid leukemia (AML)
- Planned to undergo or have undergone allogeneic hematopoietic stem cell transplantation (HSCT)
- ECOG performance status of 2 or less
- Normal organ and bone marrow function as defined by specific blood counts and liver and kidney function tests
- Left ventricular ejection fraction (LVEF) of at least 40% by MUGA scan or echocardiogram
- Negative pregnancy test for women of childbearing potential
- Agreement to use effective contraception during treatment and for 90 days after
- Ability to understand and sign informed consent
You will not qualify if you...
- Prior allogeneic hematopoietic stem cell transplant
- Relapsed or refractory disease as shown by recent bone marrow evaluation
- History of other malignancies except if disease-free for 5 years or limited to certain skin cancers or cervical cancer in situ
- Active hepatitis B or C infection
- Congestive heart failure New York Heart Association class 3 or 4 or heart dysfunction with LVEF below 40%
- History of life-threatening arrhythmias or long-QT syndrome
- QTc interval 450 ms or greater or other risk factors for QT prolongation
- Uncontrolled illness limiting study compliance
- Active infection requiring intravenous antibiotics within 7 days before treatment start
- Acute graft versus host disease requiring high-dose steroids or escalating immunosuppression
- Use of investigational agents not approved for any indication during the study
- Evidence of residual or relapsed disease before treatment start
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 24 months
Participants take ivosidenib or placebo tablets orally once daily in continuous 28-day cycles to prevent relapse of AML after stem cell transplantation.
Regular study visits and assessments throughout treatment period
Duration - Up to 12 months
Participants are followed for additional monitoring after treatment ends to assess long-term outcomes and safety.
Study visits during follow-up period
Trial Site Locations
Total: 4 locations
1
Emory University Hospital
Atlanta, Georgia, United States, 30322
Actively Recruiting
2
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
3
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Actively Recruiting
4
Froedtert Hospital & the Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
Research Team
A
Amir T Fathi, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
SINGLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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