Ivosidenib as Maintenance Therapy in Transplant-Ineligible IDH1-mutated AML and HR-MDS

NCT07607418 | Recruiting Phase 2

• 1 other identifier: 2026 (38)
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

AML (Acute Myeloid Leukemia); MDS (Myelodysplastic Syndrome)

Outcome Measures

Primary Outcomes (1)

• Disease free survival (DFS)

  The time from surgery to the onset of tumor recurrence or death from any cause.

  12 months

Secondary Outcomes (3)

• Complete response (CR)

  4 months

• Overall survival (OS)

  36 months

• Adverse events

  12 months

Study Arms (1)

Ivosidenib

EXPERIMENTAL

Drug: Ivosidenib

Interventions

Ivosidenib DRUG

Ivosidenib: 500 mg, PO, QD, 28-day cycles

Ivosidenib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Ruijin Hospital: lead

Study Sites (1)

Ruijin Hospital

Shanghai, Shanghai Municipality, 200025, China

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Anemia, Refractory, with Excess of Blasts

Interventions

ivosidenib

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Anemia, Refractory; Anemia; Myelodysplastic Syndromes; Bone Marrow Diseases

Central Study Contacts

Sujiang Zhang

CONTACT

86-21-64370045 ext 788755; zbruce.zhang@hotmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of the Department of Hematology, Ruijin Hospital North

Study Record Dates

• First Submitted: May 19, 2026
• First Posted: May 26, 2026
• Study Start: May 22, 2026
• Primary Completion (Estimated): May 30, 2027
• Study Completion (Estimated): December 30, 2027
• Last Updated: May 26, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)