Acute Myeloid LeukemiaPhase 2NCT07007949
a New Treatment of Newly Diagnosed IDH1 Mutation Acute Myeloid Leukemia
A Phase 2 study testing Ivosidenib combined with venetoclax and azacitidine in people with Acute Myeloid Leukemia and IDH1 Gene Mutation, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
This is a single arm, open-label, multicenter clinical trial to evaluate the efficacy and safety of ivosidenib+venetoclax+ azacitidine in adult Chinese subjects with newly diagnosed IDH1m AML.A total of approximately 42 China Nationwide subjects with newly diagnosed IDH1m AML will participate in the study.The primary… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, IDH1 Gene Mutation, Ivosidenib
- What is being tested?
- Ivosidenib combined with venetoclax and azacitidine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2025to 31 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- Be ≥18 years of age
- Have previously untreated AML, defined according to World Health Organization criteria. Subjects with extramedullary disease alone (ie, no detectable bone marrow and no detectable peripheral blood AML) are not eligible for the study.
- Have an IDHl mutation resulting in an R132C, R132G, R132H, R132L, or R132S substitution.
- Have an ECOG PS score of 0 to 2.
- Have adequate hepatic function, as evidenced by:
- Serum total bilirubin ≤2 × ULN, unless considered to be due to Gilbert's disease or underlying leukemia, where it must be <3 x ULN.
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- Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤3.0 × ULN, unless considered to be due to underlying leukemia.
- Have adequate renal function, as evidenced by serum creatinine ≤2.0 x ULN or creatinine clearance >30 mL/min based on the Cockcroft-Gault glomerular filtration rate.
- Have agreed to undergo serial blood and bone marrow sampling.
- Be able to understand and willing to sign an informed consent form.
- Be willing to complete QoL assessments during study treatment and at the designated time points following treatment discontinuation.
- If female with reproductive potential, must have a negative serum pregnancy test prior to the start of study therapy. Female subjects with reproductive potential are defined as sexually mature women who have not undergone a hysterectomy, bilateral oophorectomy, or tubal occlusion or who have not been naturally postmenopausal for at least 24 consecutive months. Females of reproductive potential, as well as fertile men with female partners of reproductive potential, must use 2 effective forms of contraception (including at least 1 barrier form) from the time of giving informed consent throughout the study and for 90 days (both females and males) following the last dose of study drugs). Effective forms of contraception are defined as hormonal oral contraceptives, injectables, patches, intrauterine devices, intrauterine hormone-releasing systems, bilateral tubal ligation, condoms with spermicide, or male partner sterilization.
Who cannot join
- Have received any prior treatment for AML with the exception of nononcolytic treatments to stabilize disease such as hydroxyurea or leukapheresis.
- Have received a hypomethylating agent for myelodysplastic syndrome (MDS).
- Subject has favorable risk cytogenetics such as t(8;21), inv(16), t(16;16) or t(15;17).
- Subject has acute promyelocytic leukemia
- Subjects who had previously received treatment for an antecedent hematologic disorder, including investigational agents, may not be randomized until a washout period of at least 5 half-lives of the investigational agent has elapsed since the last dose of that agent.
- Have received prior treatment with an IDH1 inhibitor or BCL-2 inhibitor.
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- Have a known hypersensitivity to any of the components of Ivosidenib, venetoclax, or azacitidine.
- Are female and pregnant or breastfeeding.
- Are taking known strong cytochrome P450 (CYP) 3A4 inducers or sensitive CYP3А4 substrate medications with a narrow therapeutic window, unless they can be transferred to other medications within ≥5 half-lives prior to dosing.
- Have an active, uncontrolled, systemic fungal, bacterial, or viral infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment.
- Have a prior history of malignancy other than MDS or myeloproliferative disorder, unless the subject has been free of the disease for ≥1 year prior to the start of study treatment. However, subjects with the following history/concurrent conditions or similar indolent cancer are allowed to participate in the study:
- Basal or squamous cell carcinoma of the skin
- Carcinoma in situ of the cervix
- Have had significant active cardiac disease within 6 months prior to the start of study treatment, including New York Heart Association Class (NYHA) Class III or IV congestive heart failure, myocardial infarction, unstable angina, and/or stroke.
- Have a heart-rate corrected QT interval using Fridericia's method (QTcF) >470 msec or any other factor that increases the risk of QT prolongation or arrhythmic events (eg, NYHA Class III or IV congestive heart failure, hypokalemia, family history of long QT interval syndrome). Subjects with prolonged QTcF interval in the setting of bundle branch block may participate in the study.
- Have a known infection caused by human immunodeficiency virus or active hepatitis B virus (HBV) or hepatitis C virus that cannot be controlled by treatment.
- Have dysphagia, short-gut syndrome, gastroparesis, or any other condition that limits the ingestion or gastrointestinal absorption of orally administered drugs.
- Have uncontrolled hypertension (systolic blood pressure [BP] >180 mmHg or diastolic BP>100 mmHg).
- Have clinical symptoms suggestive of active central nervous system (CNS) leukemia or known CNS leukemia. Evaluation of cerebrospinal fluid during Screening is only required if there is a clinical suspicion of CNS involvement by leukemia during Screening.
- Have immediate, life-threatening, severe complications of leukemia, such as uncontrolled bleeding, pneumonia with hypoxia or sepsis, and/or disseminated intravascular coagulation.
- Have any other medical or psychological condition deemed by the Investigator to be likely to interfere with the subject's ability to give informed consent or participate in the study.
- Are taking medications that are known to prolong the QT interval unless they can be transferred to other medications within ≥5 half-lives prior to dosing, or unless the medications can be properly monitored during the study. (If equivalent medication is not available, heart rate corrected QT interval [QTc] will be closely monitored.)
- Subjects with a known medical history of progressive multifocal leukoencephalopathy.
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
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
Su-ning Chen, M.D., +8613814881746, chensuning@sina.com
About this study
Official title: Ivosidenib Combined With Venetoclax and Azacitidine for the Treatment of Newly Diagnosed IDH1 Mutation Acute Myeloid Leukemia: a Prospective, Single-arm, Two-cohorts, Study
This is a single arm, open-label, multicenter clinical trial to evaluate the efficacy and safety of ivosidenib+venetoclax+ azacitidine in adult Chinese subjects with newly diagnosed IDH1m AML.A total of approximately 42 China Nationwide subjects with newly diagnosed IDH1m AML will participate in the study.The primary endpoint of the study is the complete remission(CR) + CR with partial hematologic recovery(CRh) rate, and the key secondary endpoints are CR rate,event-free survival (EFS),overall survival (OS),the objective response rate (ORR).
- Conditions
- Acute Myeloid Leukemia, IDH1 Gene Mutation, Ivosidenib
- Being tested
- Ivosidenib combined with venetoclax and azacitidine
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ivosidenib combined with venetoclax and azacitidineDrug
Also called Unfit Arm
- Ivosidenib (Ivo): The dosage is 500 mg, administered orally once daily (QD). Oral administration begins on Day 15 of Cycle 1 (C1D15) and continues on each subsequent day of the following cycles. Each cycle lasts 28 days (±2 days), with continuous dosing. • Venetoclax (Ven): The dosage is 100 mg on Day 1 of Cycle 1 (C1D1), 200 mg on Day 2 of Cycle 1 (C1D2), and 400 mg on Days 3-14 of Cycle 1 (C1D3-14), administered orally once daily (QD). For subsequent cycles, the dosage is 400 mg on Days 1-14, administered orally once daily (QD). • Azacitidine (Aza): The dosage is 75 mg/m² per day, administered via subcutaneous injection (Subcutaneous injection, SC) or intravenous injection (Intravenous, IV). It is given during the first week (7 days) of each 4-week (28-day) cycle (or according to the 5-2-2 dosing schedule). Whenever possible, each subject should use the same dosing schedule throughout the treatment period.
Ivosidenib combined with venetoclax and azacitidineDrug
Also called Fit Arm
- Ivosidenib (Ivo): The dosage is 500 mg, administered orally once daily (QD). Oral administration begins on Day 15 of Cycle 1 (C1D15) and continues on each subsequent day of the following cycles. Each cycle lasts 28 days (±2 days), with continuous dosing. • Venetoclax (Ven): The dosage is 100 mg on Day 1 of Cycle 1 (C1D1), 200 mg on Day 2 of Cycle 1 (C1D2), and 400 mg on Days 3-14 of Cycle 1 (C1D3-14), administered orally once daily (QD). For subsequent cycles, the dosage is 400 mg on Days 1-14, administered orally once daily (QD). • Azacitidine (Aza): The dosage is 75 mg/m² per day, administered via subcutaneous injection (Subcutaneous injection, SC) or intravenous injection (Intravenous, IV). It is given during the first week (7 days) of each 4-week (28-day) cycle (or according to the 5-2-2 dosing schedule). Whenever possible, each subject should use the same dosing schedule throughout the treatment period.
| Study group | Role | What they receive |
|---|---|---|
| Unfit Arm | Experimental | Unfit subjects should be treated for a minimum of 6 cycles of combination therapy unless they experience relapse after achieving a CR, CRi (including CRp), or morphologic leukemia-free state (MLFS); disease progression after having previously attained partial remission (PR) or stable disease; unacceptable toxicity (adverse event [AE]); confirmed pregnancy; withdrawal by subject; protocol violation; death; end of Study or other Protocol-specified event endpoints (refer to study design). Receives: Drug: Ivosidenib combined with venetoclax and azacitidine |
| Fit Arm | Experimental | Fit subjects should be treated for a minimum of 2 cycles of combination therapy unless they experience relapse after achieving a CR, CRi (including CRp), or morphologic leukemia-free state (MLFS); disease progression after having previously attained partial remission (PR) or stable disease; unacceptable toxicity (adverse event [AE]); confirmed pregnancy; withdrawal by subject; protocol violation; death; end of Study or other Protocol-specified event endpoints (refer to study design). Receives: Drug: Ivosidenib combined with venetoclax and azacitidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
CR + CRh rate
CR is defined as Bone marrow blasts <5% and no Auer rods; absence of extramedullary disease; ANC ≥1.0 × 109/L (1000/µL); platelet count ≥100 × 109/L (100,000/µL); independence of red blood cell transfusions.
CRh is defined as a CR with partial recovery of peripheral blood counts where absolute neutrophil count (ANC) is > 0.5 × 109/L [500/µL], and platelet count is > 50 × 109/L [50,000/µL].
Measured: 1 year
Secondary outcomes (4)
CR rate
CR is defined as bone marrow blasts < 5% and no Auer rods, absence of extramedullary disease, ANC ≥ 1.0 × 109/L [1000/µL], platelet count ≥ 100 × 109/L [100,000/µL], and independence of RBC transfusions).
Measured: 1 year
EFS
Event-free survival (EFS) is defined as the time from Cycle 1, Day 1 (C1D1) until treatment failure, relapse from remission, or death from any cause, whichever occurs first. Treatment failure is defined as failure to achieve CR by Week 24.
Measured: 1 year
OS
Overall survival (OS) is defined as the time from date of C1D1 to the date of death due to any cause.
Measured: 1 year
ORR
Objective response rate (ORR) is defined as the rate of CR, CRi (including complete remission with incomplete platelet recovery [CRp], PR, and MLFS.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2025
- Study start1 Jun 2025 (estimated)
- Primary completion30 Apr 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital of Soochow UniversityThe 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
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chen Suning, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07007949
- Sponsor's protocol ID
- IVO01
- Status last verified
- May 2025
- First submitted
- 9 May 2025
- Last updated
- 6 Jun 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ivosidenib Combined With Venetoclax and Azacitidine for the Treatment of Newly Diagnosed IDH1 Mutation Acute Myeloid Leukemia: a Prospective, Single-arm, Two-cohorts, Study
- ClinicalTrials.gov ID
- NCT07007949
- Acronym / sponsor ID
- IVO01
- Conditions
- Acute Myeloid Leukemia, IDH1 Gene Mutation, Ivosidenib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 1 Jun 2025 (estimated)
- Primary completion
- 30 Apr 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 6 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single arm, open-label, multicenter clinical trial to evaluate the efficacy and safety of ivosidenib+venetoclax+ azacitidine in adult Chinese subjects with newly diagnosed IDH1m AML.A total of approximately 42 China Nationwide subjects with newly diagnosed IDH1m AML will participate in the study.The primary endpoint of the study is the complete remission(CR) + CR with partial hematologic recovery(CRh) rate, and the key secondary endpoints are CR rate,event-free survival (EFS),overall survival (OS),the objective response rate (ORR).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Unfit Arm | Experimental | Drug: Ivosidenib combined with venetoclax and azacitidineUnfit subjects should be treated for a minimum of 6 cycles of combination therapy unless they experience relapse after achieving a CR, CRi (including CRp), or morphologic leukemia-free state (MLFS); disease progression after having previously attained partial remission (PR) or stable disease; unacceptable toxicity (adverse event [AE]); confirmed pregnancy; withdrawal by subject; protocol violation; death; end of Study or other Protocol-specified event endpoints (refer to study design). |
| Fit Arm | Experimental | Drug: Ivosidenib combined with venetoclax and azacitidineFit subjects should be treated for a minimum of 2 cycles of combination therapy unless they experience relapse after achieving a CR, CRi (including CRp), or morphologic leukemia-free state (MLFS); disease progression after having previously attained partial remission (PR) or stable disease; unacceptable toxicity (adverse event [AE]); confirmed pregnancy; withdrawal by subject; protocol violation; death; end of Study or other Protocol-specified event endpoints (refer to study design). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Be ≥18 years of age
- Have previously untreated AML, defined according to World Health Organization criteria. Subjects with extramedullary disease alone (ie, no detectable bone marrow and no detectable peripheral blood AML) are not eligible for the study.
- Have an IDHl mutation resulting in an R132C, R132G, R132H, R132L, or R132S substitution.
- Have an ECOG PS score of 0 to 2.
- Have adequate hepatic function, as evidenced by:
Key exclusion criteria (23 listed)
- Have received any prior treatment for AML with the exception of nononcolytic treatments to stabilize disease such as hydroxyurea or leukapheresis.
- Have received a hypomethylating agent for myelodysplastic syndrome (MDS).
- Subject has favorable risk cytogenetics such as t(8;21), inv(16), t(16;16) or t(15;17).
- Subject has acute promyelocytic leukemia
- Subjects who had previously received treatment for an antecedent hematologic disorder, including investigational agents, may not be randomized until a washout period of at least 5 half-lives of the investigational agent has elapsed since the last dose of that agent.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ivosidenib combined with venetoclax and azacitidineAlso: Unfit Arm• Ivosidenib (Ivo): The dosage is 500 mg, administered orally once daily (QD). Oral administration begins on Day 15 of Cycle 1 (C1D15) and continues on each subsequent day of the following cycles. Each cycle lasts 28 days (±2 days), with continuous dosing. • Venetoclax (Ven): The dosage is 100 mg on Day 1 of Cycle 1 (C1D1), 200 mg on Day 2 of Cycle 1 (C1D2), and 400 mg on Days 3-14 of Cycle 1 (C1D3-14), administered orally once daily (QD). For subsequent cycles, the dosage is 400 mg on Days 1-14, administered orally once daily (QD). • Azacitidine (Aza): The dosage is 75 mg/m² per day, administered via subcutaneous injection (Subcutaneous injection, SC) or intravenous injection (Intravenous, IV). It is given during the first week (7 days) of each 4-week (28-day) cycle (or according to the 5-2-2 dosing schedule). Whenever possible, each subject should use the same dosing schedule throughout the treatment period. | Drug | Unfit Arm |
| Ivosidenib combined with venetoclax and azacitidineAlso: Fit Arm• Ivosidenib (Ivo): The dosage is 500 mg, administered orally once daily (QD). Oral administration begins on Day 15 of Cycle 1 (C1D15) and continues on each subsequent day of the following cycles. Each cycle lasts 28 days (±2 days), with continuous dosing. • Venetoclax (Ven): The dosage is 100 mg on Day 1 of Cycle 1 (C1D1), 200 mg on Day 2 of Cycle 1 (C1D2), and 400 mg on Days 3-14 of Cycle 1 (C1D3-14), administered orally once daily (QD). For subsequent cycles, the dosage is 400 mg on Days 1-14, administered orally once daily (QD). • Azacitidine (Aza): The dosage is 75 mg/m² per day, administered via subcutaneous injection (Subcutaneous injection, SC) or intravenous injection (Intravenous, IV). It is given during the first week (7 days) of each 4-week (28-day) cycle (or according to the 5-2-2 dosing schedule). Whenever possible, each subject should use the same dosing schedule throughout the treatment period. | Drug | Fit Arm |
Primary outcome
CR + CRh rate
Time frame: 1 year
CR is defined as Bone marrow blasts <5% and no Auer rods; absence of extramedullary disease; ANC ≥1.0 × 109/L (1000/µL); platelet count ≥100 × 109/L (100,000/µL); independence of red blood cell transfusions. CRh is defined as a CR with partial recovery of peripheral blood counts where absolute neutrophil count (ANC) is > 0.5 × 109/L [500/µL], and platelet count is > 50 × 109/L [50,000/µL].
4 secondary outcomes
CR rate
Time frame: 1 year
EFS
Time frame: 1 year
OS
Time frame: 1 year
ORR
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jun 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ivosidenib combined with venetoclax and azacitidine in people with Acute Myeloid Leukemia and IDH1 Gene Mutation, run by The First Affiliated Hospital of Soochow University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Su-ning Chen, M.D. (008613814881746, chensuning@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
CR + CRh rate (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jun 2025.