Acute Myeloid LeukemiaPhase 2NCT05401097
IDH Targeted/Non- Targeted vs Non-targeted/IDH-targeted Approaches in the Treatment of Newly Diagnosed IDH Mutated AML Patients Not Candidates for Intensive Induction Therapy (I- DATA Study)
A Phase 2 study testing Azacitidine, Biopsy and Enasidenib in people with Acute Myeloid Leukemia, run by Alice Mims at 4 sites in United States.
Trial summary
This phase II study compares the order of treatment with ivosidenib or enasidenib and azacitidine plus venetoclax in treating older patients with acute myeloid leukemia with genetic changes in the IDH1 or IDH2 genes (IDH mutated). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Azacitidine, Biopsy and Enasidenib
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 125 participants
- Where
- 4 sitesUnited States
- Study dates
- 13 Sep 2022to 30 Jun 2029
- 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
- Patients with newly diagnosed IDH1 or IDH2 mutated AML
- Not a candidate for or refuses intensive induction therapy
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
- Creatinine clearance > 40 ml/min
- Aspartate aminotransferase (AST)/ alanine aminotransferase (ALT) < 5 x upper limit of normal
- Total bilirubin < 1.5 x upper limit of normal (except for patients with Gilbert's disease)
Show all 29Show fewer
- At the time of Venetoclax initiation, white blood cells (WBC) needs to be < 25 × 103 microliter: Hydroxyurea can be used to achieve that level.
- For female patients of childbearing potential, willingness to abstain from heterosexual intercourse or use a protocol-recommended method of contraception from the screening visit throughout the study treatment period and for 6 months following the last dose of either study drug. A serum pregnancy test will be done at screening. A serum or urine pregnancy test will be done on Day 1 of each cycle for women of childbearing potential. If the urine pregnancy test is positive, a serum pregnancy test must be performed per institutional standards.
- The following methods are acceptable methods of contraception for the purpose of this study:
- Highly Effective Contraception Methods that can achieve a failure rate of less than 1% per year when used consistently and correctly are considered as highly effective birth control methods (Clinical Trials Facilitation Group 2014):
- Combined (estrogen and progestin containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal).
- Progestin-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable).
- Intrauterine device.
- Intrauterine hormone-releasing system.
- Bilateral tubal occlusion.
- Vasectomized partner, provided that partner is the sole sexual partner of the female study participant and that the vasectomized partner has received medical assessment of the surgical success.
- Sexual abstinence- only if defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the patient.
- Acceptable Birth Control Methods that are not Highly Effective Contraception Acceptable birth control methods that result in a failure rate of more than 1% per year:
- Progestin-only oral hormonal contraception, where inhibition of ovulation is not the primary mode of action.
- Male or female condom with or without spermicide.
- Cap, diaphragm, or sponge with spermicide. A combination of male condom with either cap, diaphragm, or sponge with spermicide (double barrier methods) are also acceptable, but not highly effective, birth control methods.
- The following methods are NOT acceptable methods of contraception for the purpose of this study:
- Periodic abstinence (calendar, symptothermal, postovulation methods).
- Withdrawal (coitus interruptus).
- Spermicides only.
- Lactational amenorrhea method.
- Combination of male and female condom
- For male patients of childbearing potential having intercourse with females of childbearing potential, the willingness to abstain from heterosexual intercourse or use a protocol recommended method of contraception from the screening visit throughout the study treatment period and for 3 months following the last dose of either study drug. Males must also refrain from sperm donation from the screening visit throughout the study treatment period and for 3 months following the last dose of either dose of study drug
- Willingness to comply with scheduled visits, drug administration plan, imaging studies, laboratory tests, other study procedures and study restrictions
Who cannot join
- Patients with acute promyelocytic leukemia
- Known active central nervous system involvement of leukemia
- Any active malignancy requiring therapy, that would interfere with the interpretation of study endpoints.
- Evidence of ongoing uncontrolled systemic bacterial, fungal or viral infection at the time of start of study treatment
- Uncontrolled infection with hepatitis C, hepatitis B, or human immunodeficiency virus (HIV)
- Pregnancy or breast feeding
- Concurrent participation in an investigational drug trial with therapeutic intent defined as prior study therapy within 14 days prior to study treatment
- Inability to tolerate oral medications including symptomatic disease significantly affecting gastrointestinal function such as inflammatory bowel disease or resection of stomach or small bowel
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
The Ohio State University Comprehensive Cancer Center contact
United States4 sites, 4 recruiting
- Recruiting
University of Colorado
Aurora, Colorado, 80205
Derek Schatz, 303-724-9562, derek.schatz@cuanschutz.edu
Mathew Angelos, MD, PhD
- Recruiting
UNC Hospitals, University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514
Study Coordinator, 919-966-4432, cancerclinicaltrials@med.unc.edu
Joshua Zeidner, MD
- Recruiting
Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210
Molly Brandenburg, 614-685-9573, molly.brandenburg@osumc.edu
Alice S. Mims, MD
- Recruiting
UT Southwestern Medical Center at Dallas
Dallas, Texas, 75235
Donglan Xia, RN, PhD, 469-852-0680, Donglan.Xia@utsouthwestern.edu
Yazan Madanat, MD
About this study
Official title: Randomized, Sequential, Open-Label Study to Evaluate the Efficacy of IDH Targeted/Non- Targeted Versus Non-targeted/IDH-targeted Approaches in the Treatment of Newly Diagnosed IDH Mutated AML Patients Not Candidates for Intensive Induction Therapy (I- DATA Study)
This phase II study compares the order of treatment with ivosidenib or enasidenib and azacitidine plus venetoclax in treating older patients with acute myeloid leukemia with genetic changes in the IDH1 or IDH2 genes (IDH mutated). Ivosidenib is in a class of medications called isocitrate dehydrogenase-1 (IDH1) inhibitors. It works by slowing or stopping the growth of cancer cells. Enasidenib is in a class of medications called an IDH2 inhibitor. It also works by slowing or stopping the growth of cancer cells. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Azacitidine is in a class of medications called demethylation agents. It works by helping the bone marrow to produce normal blood cells and by killing abnormal cells. This study may help researchers determine which treatment order is best for older patients with IDH mutated acute myeloid leukemia: 1) ivosidenib or enasidenib followed by azacitidine plus venetoclax; or 2) azacitidine plus venetoclax followed by ivosidenib or enasidenib.
Read the detailed description
PRIMARY OBJECTIVE:
- To compare overall treatment failure at 24 months in newly diagnosed IDH1 or IDH2 mutated AML patients ≥18 years who are not candidates for intensive induction chemotherapy randomized to either sequential treatment with an IDH inhibitor in combination with azacitidine followed by venetoclax in combination with azacitidine (Arm A) or sequential treatment with venetoclax in combination with azacitidine followed by an IDH inhibitor in combination with azacitidine (Arm B).
SECONDARY OBJECTIVES:
- To compare overall survival at 24 months between patients treated on the two sequential treatment arms.
II. To compare time to overall treatment failure and time-to-event overall survival between patients treated on the two sequential treatment arms.
III. To determine the degree of response and compare complete remission (CR) rates, CR/complete remission with hematologic improvement (CRh)/complete remission with incomplete blood count recovery (CRi) rates, and overall response rates (CR/CRh/CRi/morphologic leukemia free state [MLFS]) for first-line therapy and second-line therapy between patients treated on the two sequential treatment arms.
IV. To compare the duration of response (CR/CRh/CRi) to first-line therapy and second-line therapy between patients treated on the two sequential treatment arms.
- To determine toxicity profiles for patients treated on the two sequential treatment arms, overall and by first-line treatment and by second-line treatment.
VI. To determine causes that would not allow patients that first line treatment fails to go on to second line treatment.
VII. To determine the number and proportion of patients who are able to go onto allogeneic transplantation in both treatment arms.
EXPLORATORY OBJECTIVES:
- To assess the clonal, biochemical and differentiation changes in AML cells during IDH-inhibitor and venetoclax treatment using flow cytometry and serial next generation sequencing on bone marrow specimens before and during treatment to assess for potential resistance mutations or clonal evolution that may be predictors of relapse.
II. To examine molecular properties of AML cells associated with primary and secondary resistance to each treatment arm to determine if particular subtypes of AML may be more or less likely to respond to a certain treatment modality.
III. To perform minimal residual disease (MRD) monitoring via liquid biopsy via our custom-designed 30-gene mutation ArcherPlex panel to monitor clonal dynamics during both sequential treatment arms.
IV. To determine the feasibility of longitudinal sociodemographic and social determinants of health (SDOH) data collection for adults with AML on a clinical trial.
- To determine the acceptability of longitudinal sociodemographic/SDOH data collection for adults with AML on a clinical trial.
VI. To describe changes in SDOH for adults with AML on a clinical trial. VII. To describe associations between SDOH measures and trial outcomes for adults with AML on a clinical trial.
OUTLINE: Patients are randomized into 1 of 2 arms.
ARM A: For IDH1 mutated AML patients randomized to first-line therapy with IDHi+aza, patients will receive Ivosidenib 500mg po orally daily on Days 1-28 of each 28 day cycle. For IDH2 mutated AML patients randomized to first-line therapy with IDHi+aza, patients will receive Enasidenib 100mg po orally daily on Days 1-28 of each 28 day cycle. Azacitidine will be given to both groups intravenously or subcutaneously at 75mg/m2 daily on days 1-7, or 1-5/8-9, or days 1-4/7-9 of each 28 day cycle. Subsequent cycles after CR/CRi/CRh/MLFS achievement may be adjusted in timing and dosing.
ARM B: For both IDH1 and IDH2 mutated AML patient randomized to first-line therapy with Ven+aza, patients will receive venetoclax dosing with the ramp-up and dosing per the FDA-label (based off of concurrent drug interactions). Azacitidine will be given intravenously at 75mg/m2 daily on days 1-7, or 1-5/8-9, or days 1-4/7-9 of each 28-day cycle. Subsequent cycles after CR/CRi/CRh/MLFS achievement may be adjusted in timing and dosing.
After completion of the study treatment, patients are followed up at 30 days and then every 3 months for 5 years from registration, until death, or withdrawal of consent from study assessments and all further follow-up.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Azacitidine, Biopsy, Enasidenib, Ivosidenib, Venetoclax
- 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.
AzacitidineDrug
Also called 5 AZC, 5-AC, 5-Azacytidine, 5-AZC
Given IV or SC
BiopsyProcedure or surgery
Also called BIOPSY_TYPE, Bx
Undergo biopsy of the bone marrow
EnasidenibDrug
Also called AG-221, CC-90007 Free Base
Given PO
IvosidenibDrug
Also called AG-120, Tibsovo
Given PO
VenetoclaxDrug
Also called ABT-0199, ABT-199, ABT199, GDC-0199
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Arm A (IDHi+Aza followed by Ven+aza) | Experimental | For IDH1 mutated AML patients randomized to first-line therapy with IDHi+aza, patients will receive Ivosidenib 500mg po orally daily on Days 1-28 of each 28 day cycle. For IDH2 mutated AML patients randomized to first-line therapy with IDHi+aza, patients will receive Enasidenib 100mg po orally daily on Days 1-28 of each 28 day cycle. Azacitidine will be given to both groups intravenously or subcutaneously at 75mg/m2 daily on days 1-7, or 1-5/8-9, or days 1-4/7-9 of each 28-day cycle.Subsequent cycles after CR/CRi/CRh/MLFS achievement may be adjusted in timing and dosing. Receives: Drug: Azacitidine; Procedure: Biopsy; Drug: Enasidenib; Drug: Ivosidenib; Drug: Venetoclax |
| Arm B (Ven+aza followed by IDHi+aza) | Experimental | For both IDH1 and IDH2 mutated AML patient randomized to first-line therapy with Ven+aza, patients will receive venetoclax dosing with the ramp-up and dosing per the FDA-label (based off of concurrent drug interactions). Azacitidine will be given intravenously at 75mg/m2 daily on days 1-7, or 1-5/8-9, or days 1-4/7-9 of each 28-day cycle. Subsequent cycles after CR/CRi/CRh/MLFS achievement may be adjusted in timing and dosing. Receives: Drug: Azacitidine; Procedure: Biopsy; Drug: Enasidenib; Drug: Ivosidenib; Drug: Venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall treatment failure
Defined as: 1) second occurrence of any of disease progression, relapse, failure to achieve complete remission (CR)/complete remission with hematologic improvement (CRh)/complete remission with incomplete blood count recovery (CRi), or 2) death from any cause. Within each treatment sequence, overall treatment failure rate will be defined as the number of patients with events divided by the number of eligible patients randomized. Patients who go to transplant will be considered a treatment success for a particular treatment sequence. All randomized patients meeting the eligibility criteria will be evaluable for treatment failure status by intention to treat. Will be analyzed using a Cochran-Mantel-Haenszel test, testing for a difference in proportions and stratifying on isocitrate dehydrogenase (IDH) mutation status.
Measured: At 12 months from date of randomization
Secondary outcomes (3)
Difference in treatment failure rates between the two arms
Will use a two-sided Cochran-Mantel-Haenszel test, stratifying on IDH mutation status.
Measured: Up to 2 years
Overall survival (OS)
Will be summarized using the Kaplan-Meier method and will be compared between arms using a stratified log-rank test (stratified on IDH mutation status).
Measured: Up to 5 years
Duration of response
Will be summarized using the Kaplan-Meier method and will be compared between arms using a stratified log-rank test (stratified on IDH mutation status).
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2022
- Study start13 Sep 2022
- Primary completion30 Sep 2027 (estimated)
- Study completion30 Jun 2029 (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.
- 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
- Sequential
- Masking
- None (open label)
- Enrolment
- 125 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alice Mims
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor-investigator, Alice Mims, Ohio State University Comprehensive Cancer Center
- Investigators
- Alice S Mims, MD, Principal investigator, Ohio State University Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05401097
- Sponsor's protocol ID
- OSU-21330
- Other IDs
- NCI-2022-01324, R01CA262496
- Status last verified
- Jun 2026
- First submitted
- 16 May 2022
- Last updated
- 23 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Sequential, Open-Label Study to Evaluate the Efficacy of IDH Targeted/Non- Targeted Versus Non-targeted/IDH-targeted Approaches in the Treatment of Newly Diagnosed IDH Mutated AML Patients Not Candidates for Intensive Induction Therapy (I- DATA Study)
- ClinicalTrials.gov ID
- NCT05401097
- Acronym / sponsor ID
- OSU-21330
- Conditions
- Acute Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 125 participants (estimated)
- Lead sponsor
- Alice MimsUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 13 Sep 2022 (actual)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 30 Jun 2029 (estimated)
- First posted
- 2 Jun 2022 (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 phase II study compares the order of treatment with ivosidenib or enasidenib and azacitidine plus venetoclax in treating older patients with acute myeloid leukemia with genetic changes in the IDH1 or IDH2 genes (IDH mutated). Ivosidenib is in a class of medications called isocitrate dehydrogenase-1 (IDH1) inhibitors. It works by slowing or stopping the growth of cancer cells. Enasidenib is in a class of medications called an IDH2 inhibitor. It also works by slowing or stopping the growth of cancer cells. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Azacitidine is in a class of medications called demethylation agents. It works by helping the bone marrow to produce normal blood cells and by killing abnormal cells. This study may help researchers determine which treatment order is best for older patients with IDH mutated acute myeloid leukemia: 1) ivosidenib or enasidenib followed by azacitidine plus venetoclax; or 2) azacitidine plus venetoclax followed by ivosidenib or enasidenib.
Detailed description
PRIMARY OBJECTIVE:
- To compare overall treatment failure at 24 months in newly diagnosed IDH1 or IDH2 mutated AML patients ≥18 years who are not candidates for intensive induction chemotherapy randomized to either sequential treatment with an IDH inhibitor in combination with azacitidine followed by venetoclax in combination with azacitidine (Arm A) or sequential treatment with venetoclax in combination with azacitidine followed by an IDH inhibitor in combination with azacitidine (Arm B).
SECONDARY OBJECTIVES:
- To compare overall survival at 24 months between patients treated on the two sequential treatment arms.
II. To compare time to overall treatment failure and time-to-event overall survival between patients treated on the two sequential treatment arms.
III. To determine the degree of response and compare complete remission (CR) rates, CR/complete remission with hematologic improvement (CRh)/complete remission with incomplete blood count recovery (CRi) rates, and overall response rates (CR/CRh/CRi/morphologic leukemia free state [MLFS]) for first-line therapy and second-line therapy between patients treated on the two sequential treatment arms.
IV. To compare the duration of response (CR/CRh/CRi) to first-line therapy and second-line therapy between patients treated on the two sequential treatment arms.
- To determine toxicity profiles for patients treated on the two sequential treatment arms, overall and by first-line treatment and by second-line treatment.
VI. To determine causes that would not allow patients that first line treatment fails to go on to second line treatment.
VII. To determine the number and proportion of patients who are able to go onto allogeneic transplantation in both treatment arms.
EXPLORATORY OBJECTIVES:
- To assess the clonal, biochemical and differentiation changes in AML cells during IDH-inhibitor and venetoclax treatment using flow cytometry and serial next generation sequencing on bone marrow specimens before and during treatment to assess for potential resistance mutations or clonal evolution that may be predictors of relapse.
II. To examine molecular properties of AML cells associated with primary and secondary resistance to each treatment arm to determine if particular subtypes of AML may be more or less likely to respond to a certain treatment modality.
III. To perform minimal residual disease (MRD) monitoring via liquid biopsy via our custom-designed 30-gene mutation ArcherPlex panel to monitor clonal dynamics during both sequential treatment arms.
IV. To determine the feasibility of longitudinal sociodemographic and social determinants of health (SDOH) data collection for adults with AML on a clinical trial.
- To determine the acceptability of longitudinal sociodemographic/SDOH data collection for adults with AML on a clinical trial.
VI. To describe changes in SDOH for adults with AML on a clinical trial. VII. To describe associations between SDOH measures and trial outcomes for adults with AML on a clinical trial.
OUTLINE: Patients are randomized into 1 of 2 arms.
ARM A: For IDH1 mutated AML patients randomized to first-line therapy with IDHi+aza, patients will receive Ivosidenib 500mg po orally daily on Days 1-28 of each 28 day cycle. For IDH2 mutated AML patients randomized to first-line therapy with IDHi+aza, patients will receive Enasidenib 100mg po orally daily on Days 1-28 of each 28 day cycle. Azacitidine will be given to both groups intravenously or subcutaneously at 75mg/m2 daily on days 1-7, or 1-5/8-9, or days 1-4/7-9 of each 28 day cycle. Subsequent cycles after CR/CRi/CRh/MLFS achievement may be adjusted in timing and dosing.
ARM B: For both IDH1 and IDH2 mutated AML patient randomized to first-line therapy with Ven+aza, patients will receive venetoclax dosing with the ramp-up and dosing per the FDA-label (based off of concurrent drug interactions). Azacitidine will be given intravenously at 75mg/m2 daily on days 1-7, or 1-5/8-9, or days 1-4/7-9 of each 28-day cycle. Subsequent cycles after CR/CRi/CRh/MLFS achievement may be adjusted in timing and dosing.
After completion of the study treatment, patients are followed up at 30 days and then every 3 months for 5 years from registration, until death, or withdrawal of consent from study assessments and all further follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A (IDHi+Aza followed by Ven+aza) | Experimental | Drug: Azacitidine, Procedure: Biopsy, Drug: Enasidenib, Drug: Ivosidenib, Drug: VenetoclaxFor IDH1 mutated AML patients randomized to first-line therapy with IDHi+aza, patients will receive Ivosidenib 500mg po orally daily on Days 1-28 of each 28 day cycle. For IDH2 mutated AML patients randomized to first-line therapy with IDHi+aza, patients will receive Enasidenib 100mg po orally daily on Days 1-28 of each 28 day cycle. Azacitidine will be given to both groups intravenously or subcutaneously at 75mg/m2 daily on days 1-7, or 1-5/8-9, or days 1-4/7-9 of each 28-day cycle.Subsequent cycles after CR/CRi/CRh/MLFS achievement may be adjusted in timing and dosing. |
| Arm B (Ven+aza followed by IDHi+aza) | Experimental | Drug: Azacitidine, Procedure: Biopsy, Drug: Enasidenib, Drug: Ivosidenib, Drug: VenetoclaxFor both IDH1 and IDH2 mutated AML patient randomized to first-line therapy with Ven+aza, patients will receive venetoclax dosing with the ramp-up and dosing per the FDA-label (based off of concurrent drug interactions). Azacitidine will be given intravenously at 75mg/m2 daily on days 1-7, or 1-5/8-9, or days 1-4/7-9 of each 28-day cycle. Subsequent cycles after CR/CRi/CRh/MLFS achievement may be adjusted in timing and dosing. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (29 listed)
- Patients with newly diagnosed IDH1 or IDH2 mutated AML
- Not a candidate for or refuses intensive induction therapy
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
- Creatinine clearance > 40 ml/min
- Aspartate aminotransferase (AST)/ alanine aminotransferase (ALT) < 5 x upper limit of normal
Key exclusion criteria (8 listed)
- Patients with acute promyelocytic leukemia
- Known active central nervous system involvement of leukemia
- Any active malignancy requiring therapy, that would interfere with the interpretation of study endpoints.
- Evidence of ongoing uncontrolled systemic bacterial, fungal or viral infection at the time of start of study treatment
- Uncontrolled infection with hepatitis C, hepatitis B, or human immunodeficiency virus (HIV)
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 |
|---|---|---|
| AzacitidineAlso: 5 AZC, 5-AC, 5-Azacytidine, 5-AZCGiven IV or SC | Drug | Arm A (IDHi+Aza followed by Ven+aza), Arm B (Ven+aza followed by IDHi+aza) |
| BiopsyAlso: BIOPSY_TYPE, BxUndergo biopsy of the bone marrow | Procedure or surgery | Arm A (IDHi+Aza followed by Ven+aza), Arm B (Ven+aza followed by IDHi+aza) |
| EnasidenibAlso: AG-221, CC-90007 Free BaseGiven PO | Drug | Arm A (IDHi+Aza followed by Ven+aza), Arm B (Ven+aza followed by IDHi+aza) |
| IvosidenibAlso: AG-120, TibsovoGiven PO | Drug | Arm A (IDHi+Aza followed by Ven+aza), Arm B (Ven+aza followed by IDHi+aza) |
| VenetoclaxAlso: ABT-0199, ABT-199, ABT199, GDC-0199Given PO | Drug | Arm A (IDHi+Aza followed by Ven+aza), Arm B (Ven+aza followed by IDHi+aza) |
Primary outcome
Overall treatment failure
Time frame: At 12 months from date of randomization
Defined as: 1) second occurrence of any of disease progression, relapse, failure to achieve complete remission (CR)/complete remission with hematologic improvement (CRh)/complete remission with incomplete blood count recovery (CRi), or 2) death from any cause. Within each treatment sequence, overall treatment failure rate will be defined as the number of patients with events divided by the number of eligible patients randomized. Patients who go to transplant will be considered a treatment success for a particular treatment sequence. All randomized patients meeting the eligibility criteria will be evaluable for treatment failure status by intention to treat. Will be analyzed using a Cochran-Mantel-Haenszel test, testing for a difference in proportions and stratifying on isocitrate dehydrogenase (IDH) mutation status.
3 secondary outcomes
Difference in treatment failure rates between the two arms
Time frame: Up to 2 years
Overall survival (OS)
Time frame: Up to 5 years
Duration of response
Time frame: Up to 5 years
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 23 Jun 2026.
- The JameslineLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
The Ohio State University Comprehensive Cancer Center contact
Alice S Mims, MD principal investigator
Ohio State University Comprehensive Cancer Center
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Azacitidine, Biopsy and Enasidenib in people with Acute Myeloid Leukemia, run by Alice Mims at 4 sites in United States.
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 29 inclusion and 8 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 23 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. 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 The Ohio State University Comprehensive Cancer Center (800-293-5066, OSUCCCClinicaltrials@osumc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall treatment failure (time frame: At 12 months from date of randomization). 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 23 Jun 2026.