Tests ivosidenib as ongoing treatment for leukemia and MDS with IDH1 mutation
Tests ivosidenib as ongoing treatment in adults with IDH1-mutated AML or high-risk MDS.
Plain-language title generated from the registry record
Registry title: Ivosidenib as Maintenance Therapy in Transplant-Ineligible IDH1-mutated AML and HR-MDS
Key facts
- Where
- Shanghai, ChinaSee the site
- Phase
- Phase 2
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with IDH1-mutated acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) who are in remission and cannot have a stem cell transplant. They would take ivosidenib as maintenance treatment.
- Age
- 18 years or older
- Sex
- Women and men can join.
- Disease stage
- IDH1-mutated AML or high-risk MDS, in complete or partial remission after induction therapy. Performance status (ECOG) 2 or lower.
- Earlier treatment
- Must have had induction therapy and be ineligible for allogeneic stem cell transplant. Must not have had an IDH1 inhibitor before.
- Biomarker or test result
- Molecularly confirmed IDH1 mutation.
Main reasons for not taking part
- Earlier treatment with an IDH1 inhibitor
- Acute promyelocytic leukemia (M3 subtype)
- Severe liver or kidney problems
- HIV infection, active hepatitis B or hepatitis C
- Active infection or severe heart disease
Read by machine on 9 November 2026.
Where in China
- Ruijin hospitalShanghai, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Ruijin Hospital
- Trial code
- NCT07607418
- Record updated
- 26 May 2026
- Original description
This study will explore the efficacy and safety of ivosidenib as maintenance therapy in patients with IDH1-mutated AML and high-risk MDS who are ineligible for transplantation, along with accompanying molecular biomarker research. Patients who meet the eligibility criteria will receive ivosidenib treatment until disease progression or unacceptable toxicity. This study will provide an effective maintenance treatment option for transplant-ineligible patients with IDH1-mutated AML and high-risk MDS.Read full original descriptionShow less
A Phase II, Multicenter, Open-label Clinical Study of Ivosidenib as Maintenance Therapy in Patients With IDH1-mutated AML and High-risk MDS Who Are Ineligible for Transplantation (IVORY-MAST)
This study will explore the efficacy and safety of ivosidenib as maintenance therapy in patients with IDH1-mutated AML and high-risk MDS who are ineligible for transplantation, along with accompanying molecular biomarker research. Patients who meet the eligibility criteria will receive ivosidenib treatment until disease progression or unacceptable toxicity. This study will provide an effective maintenance treatment option for transplant-ineligible patients with IDH1-mutated AML and high-risk MDS.
Inclusion: Age ≥18 years, 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 ≤2; Signed informed consent; For women of childbearing potential: must have practiced contraception for at least one month prior to screening and commit to using contraception throughout the study period and for the required period after study completion; Female participants of childbearing potential and male participants with female partners of childbearing potential must agree to use highly effective methods of contraception from the time of signing informed consent until 180 days after the last dose of the study drug.
Exclusion: Prior treatment with an IDH1 inhibitor; Major surgery within 28 days prior to the first dose, or planned surgery during the study period; Diagnosis of any other malignancy within 5 years prior to the first dose, with the exception of early-stage malignancies that have been cured by radical treatment, such as carcinoma in situ of the lung, non-melanoma skin cancer, basal cell or squamous cell carcinoma of the skin, or cervical carcinoma in situ. Whether to exclude such patients will be determined by the investigator's assessment; Severe cardiovascular disease, such as New York Heart Association (NYHA) class 2 heart failure, unstable angina, unstable arrhythmia, myocardial infarction or cerebrovascular accident within 3 months prior to randomization; Inability to swallow, intestinal obstruction, or other factors affecting drug administration or absorption; Previous concomitant treatment with both drugs of the combination therapy targeting the same pathway as in this study; Active infection or other serious comorbidities; Human immunodeficiency virus (HIV) infection or known acquired immunodeficiency syndrome (AIDS); active hepatitis B (HBV DNA≥1000 IU/ml); hepatitis C (positive for anti-HCV antibodies and HCV-RNA above the lower limit of detection of the assay); or co-infection with hepatitis B and hepatitis C; Known history of psychoactive substance abuse or drug addiction; History of allergy to the study drug or any of its excipients; Presence of any other serious physical or mental illness, or abnormal laboratory findings that may increase the risk of study participation, interfere with the study results, or make the patient unsuitable for study participation in the opinion of the investigator; Participants with any of the following criteria will be excluded from the study:; Diagnosis of M3 subtype (acute promyelocytic leukemia, APL); Presence of severe hepatic or renal dysfunction.