AML (Acute Myeloid Leukemia)Phase 2NCT07607418
Ivosidenib as Maintenance Therapy in Transplant-Ineligible IDH1-mutated AML and HR-MDS
A Phase 2 study testing Ivosidenib in people with AML (Acute Myeloid Leukemia) and MDS (Myelodysplastic Syndrome), run by Ruijin Hospital at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AML (Acute Myeloid Leukemia), MDS (Myelodysplastic Syndrome)
- What is being tested?
- Ivosidenib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 22 May 2026to 30 Dec 2027
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Participants with any of the following criteria will be excluded from the study:
- Prior treatment with an IDH1 inhibitor.
- Diagnosis of M3 subtype (acute promyelocytic leukemia, APL).
- Presence of severe hepatic or renal dysfunction.
- Active infection or other serious comorbidities.
- Major surgery within 28 days prior to the first dose, or planned surgery during the study period.
Show all 14Show fewer
- 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.
- 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.
- 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.
- 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.
- History of allergy to the study drug or any of its excipients.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Ruijin Hospital
Shanghai, Shanghai Municipality, 200025
Sujiang Zhang, 86-21-64370045 ext 788755, zbruce.zhang@hotmail.com
About this study
Official title: 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.
- Being tested
- Ivosidenib
- Medical subject headings
- Leukemia, Myeloid, Acute, Anemia, Refractory, with Excess of Blasts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IvosidenibDrug
Ivosidenib: 500 mg, PO, QD, 28-day cycles
| Study group | Role | What they receive |
|---|---|---|
| Ivosidenib | Experimental | Not described Receives: Drug: Ivosidenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease free survival (DFS)
The time from surgery to the onset of tumor recurrence or death from any cause.
Measured: 12 months
Secondary outcomes (3)
Complete response (CR)
The proportion of patients achieved complete response (CR).
Measured: 4 months
Overall survival (OS)
The time from randomization to the time of death from any cause.
Measured: 36 months
Adverse events
The severity of adverse events will be evaluated according to the NCI CTCAE 5.0 standard.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2026
- Study start22 May 2026 (estimated)
- Primary completion30 May 2027 (estimated)
- Study completion30 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML (Acute Myeloid Leukemia)Studies of the same condition, open and under way.
- More trials run by Ruijin HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zhang sujiang, Ruijin Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07607418
- Sponsor's protocol ID
- 2026 (38)
- Status last verified
- Apr 2026
- First submitted
- 19 May 2026
- Last updated
- 26 May 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No