Actively Recruiting
Ivosidenib as Maintenance Therapy in Transplant-Ineligible Patients With IDH1-mutated Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome
Led by Ruijin Hospital · Updated on 2026-05-26
20
Participants Needed
1
Research Sites
30 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of ivosidenib as a maintenance treatment for patients with IDH1-mutated acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS) who cannot undergo transplantation. This Phase II study also includes molecular biomarker research to better understand the treatment effects. The goal is to find an effective maintenance option for these transplant-ineligible patients. Participants will receive ivosidenib orally at a dose of 500 mg once daily in 28-day cycles. The treatment will continue until the disease progresses or unacceptable side effects occur. This open-label study involves only one treatment group receiving ivosidenib. Throughout the study, participants will be monitored regularly for disease progression, treatment response including complete remission, overall survival, and any adverse events. The main outcome measured is disease-free survival at 12 months. Participants will be followed for up to 36 months to assess overall survival and safety. The total duration of participation will depend on individual treatment response and tolerability.
CONDITIONS
Brief Title
Ivosidenib as Maintenance Therapy in Transplant-Ineligible IDH1-mutated AML and HR-MDS
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older, male or female
- Molecularly confirmed diagnosis of IDH1-mutated acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS)
- Achieved complete remission (CR) or partial remission (PR) after induction therapy
- Ineligible for allogeneic hematopoietic stem cell transplantation (allo-HSCT)
- Eastern Cooperative Oncology Group (ECOG) performance status score of 2 or less
- Signed informed consent
- For women of childbearing potential: practiced contraception for at least one month prior to screening and commits to contraception throughout the study and required period after
- Female participants of childbearing potential and male participants with female partners of childbearing potential must agree to use highly effective contraception from consent until 180 days after last dose
You will not qualify if you...
- Prior treatment with an IDH1 inhibitor
- Diagnosis of M3 subtype (acute promyelocytic leukemia, APL)
- Severe hepatic or renal dysfunction
- Active infection or other serious comorbid conditions
- Major surgery within 28 days before first dose or planned surgery during study
- Diagnosis of any other malignancy within 5 years before first dose, except certain early-stage malignancies as determined by investigator
- Severe cardiovascular disease such as NYHA class 2 heart failure, unstable angina, unstable arrhythmia, recent heart attack or stroke within 3 months
- HIV infection or AIDS; active hepatitis B or C infection; co-infection with hepatitis B and C
- History of psychoactive substance abuse or drug addiction
- Any other serious physical or mental illness or abnormal lab results that increase risk or interfere with study
- Inability to swallow or intestinal obstruction affecting drug intake
- Previous treatment with both drugs targeting the same pathway as this study
- History of allergy to ivosidenib or its components
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 - Repeated 28-day cycles until disease progression or discontinuation
Participants receive ivosidenib orally once daily in 28-day cycles as maintenance therapy.
Monthly visits for treatment assessment
Trial Site Locations
Total: 1 location
1
Ruijin Hospital
Shanghai, Shanghai Municipality, China, 200025
Actively Recruiting
Research Team
S
Sujiang Zhang
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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