IDH1 MutationPhase 2NCT07471841
Olutasidenib in Relapsed IDH1 Mutated AML Patients Who Have Previously Received Venetoclax
A Phase 2 study testing Olutasidenib and Azacitidine (AZA) in people with IDH1 Mutation and Relapsed / Refractory AML, run by Timothy Pardee at 1 site in United States.
Trial summary
This is a prospective, single-arm phase 2 pilot study to assess the response rate of IDH1 mutated relapsed/refractory acute myeloid leukemia (AML) patients who receive olutasidenib after progressing on venetoclax based regimens. Each cycle will last for 28 days. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- IDH1 Mutation, Relapsed / Refractory AML
- What is being tested?
- Olutasidenib and Azacitidine (AZA)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2026to 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
- Written informed consent and HIPAA authorization for release of personal health information prior to registration. NOTE: HIPAA authorization may be included in the informed consent or obtained separately.
- Age ≥ 18 years at the time of consent.
- ECOG Performance Status of ≤ 2 within 28 days prior to registration.
- Must have histologically or cytologically documented relapsed and/or refractory Acute Myeloid Leukemia (Refractory is defined as failure to achieve a CR after induction chemotherapy or a minimum of two cycles of HMA plus venetoclax)
- Acute Myeloid Leukemia with a documented IDH1 mutation.
- No more than 2 lines of prior therapy. NOTE: One line of therapy must have contained venetoclax.
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- Persisting, non-hematologic and non-infectious toxicities from prior treatment must be Grade ≤ 2. NOTE: Documentation of these criteria is required at screening.
- Demonstrate adequate organ function as defined in the table below. All screening labs to be obtained within 14 days prior to registration.
- Renal
- Calculated creatinine clearance (Cockcroft-Gault formula will be used to calculate creatinine clearance) ≥ 30 mL/min
- Hepatic
- Total bilirubin2 ≤ 2× upper limit of normal (ULN); ≤ 3 times ULN in patients with Gilbert Syndrome
- Aspartate aminotransferase (AST) ≤ 5 × ULN
- Alanine aminotransferase (ALT) ≤ 5× ULN
- Participants of childbearing potential must have a negative serum pregnancy test within 7 days prior to initiation of treatment.
- Participants of childbearing potential who are having penile-vaginal intercourse with a person able to father a child must be willing to abstain from penile-vaginal intercourse or must use an effective method(s) of contraception. A participant able to father a child who is having penile-vaginal intercourse with a person of childbearing potential must be willing to abstain from penile-vaginal intercourse or use an effective method(s) of contraception.
- Subjects with known HIV infection may be eligible if they are on effective anti-retroviral therapy with undetectable viral load within 6 months prior to registration. Testing is not required at screening.
- Subjects with known chronic hepatitis B virus (HBV) infection may be eligible if they have an undetectable HBV viral load on suppressive therapy, if indicated. Subjects with a history of hepatitis C virus (HCV) infection may be eligible if they have been treated and cured. Subjects with an HCV infection who are currently on treatment must have an undetectable HCV viral load to be eligible for this trial. Testing is not required at screening.
- As determined by the investigator or protocol designee, ability of the subject to understand and comply with study procedures for the entire length of the study.
Who cannot join
- Previous exposure to ivosidenib or any IDH1 inhibitor.
- Active infection requiring IV systemic therapy. NOTE: Subjects receiving prophylactic antibiotics (e.g., to prevent a urinary tract infection or chronic obstructive pulmonary disease exacerbation) are eligible for the study.
- Known CNS involvement with AML.
- Previous allogeneic stem cell transplant within 60 days prior to registration.
- Treatment with any investigational drug within 21 days or 2 half-life's prior to registration.
- History of severe allergic anaphylactic reactions to olutasidenib or any of their excipients.
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- Pregnant or breastfeeding. NOTE: breast milk cannot be stored for future use while the subject is being treated on study.
- Prior or concurrent malignancy whose natural history or treatment has the potential to interfere with the safety or efficacy assessment of the investigational regimen, per treating physician discretion.
- Concomitant use of known strong (e.g. phenobarbital, enzalutamide, phenytoin, rifampicin, rifabutin, rifapentine, carbamazepine, nevirapine and St John's Wort) or moderate CYP3A inducers (e.g. bosentan, efavirenz, modafinil). The required washout period prior to starting olutasidenib is 2 weeks or 5 half-lives (whichever is longer) prior to initiation of treatment.
- Concomitant use of known sensitive CYP3A substrates (e.g. Midazolam, simvastatin, fluticasone, budesonide). The required washout period prior to starting olutasidenib is 2 weeks or 5 half-lives (whichever is longer) prior to initiation of treatment.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Atrium Health Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157
Elizabeth Parke, 980-442-2000, Elizabeth.Parke@Advocatehealth.org
Timothy Pardee, MD, PhD
About this study
Official title: Pilot Single Arm Phase 2 Study of Olutasidenib in Relapsed IDH1 Mutated AML Patients Who Have Previously Received Venetoclax
This is a prospective, single-arm phase 2 pilot study to assess the response rate of IDH1 mutated relapsed/refractory acute myeloid leukemia (AML) patients who receive olutasidenib after progressing on venetoclax based regimens. Each cycle will last for 28 days. Patients will receive olutasidenib 150 mg orally twice daily Day 1 through Day 28. After 3 cycles of olutasidenib, azacitidine 75 mg/m2 given on Day 1 through Day 7 may be added at the discretion of the treating investigator if the patient has not achieved a complete remission. Subjects with at least a PR after 6 cycles of treatment will continue treatment as previously described. Subjects without at least a partial response (PR) after 6 cycles of treatment will move to long term follow up.
- Conditions
- IDH1 Mutation, Relapsed / Refractory AML
- Being tested
- Olutasidenib, Azacitidine (AZA)
- 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.
OlutasidenibDrug
Olutasidenib 150 mg orally twice a day
Azacitidine (AZA)Drug
Azacitidine 75 mg/m2 subcutaneously or IV (over 10-40 minutes)
| Study group | Role | What they receive |
|---|---|---|
| Olutasidenib and Azacitidine | Experimental | Each cycle will last for 28 days. Patients will receive olutasidenib 150 mg orally twice daily Day 1 through Day 28. After 3 cycles of olutasidenib, azacitidine 75 mg/m2 given on Day 1 through Day 7 may be added at the discretion of the treating investigator if the patient has not achieved a complete remission or for whom loss of response is suspected. Subjects with at least a PR after 6 cycles of treatment will continue treatment.. Subjects without at least a PR after 6 cycles of treatment will move to long term follow up. Receives: Drug: Olutasidenib; Drug: Azacitidine (AZA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Composite complete remission rate
Composite complete remission (CRc) rate is defined as patients that meet the criteria for CR + CRh + CRi per the modified European LeukemiaNet (mELN) 2022.
- Complete Remission (CR) is defined as absence of leukemia cells in the bone marrow (< 5% blasts), normal blood counts (absolute neutrophil count ≥ 1000/μL and platelet count ≥ 100,000/μL), and the absence of circulating blasts, and extramedullary disease
- Complete Remission with Hematologic recovery (CRh) is defined as meeting all criteria for CR as Bone marrow myeloblasts < 5%; absence of circulating blasts; absence of extramedullary disease; both ANC ≥ 0.5x109/L (500/µL) and platelet count ≥ 50×109/L (50 000/µL)
- Complete Remission with Incomplete hematologic recovery (CRi) is defined as meeting all criteria for Complete Remission (CR) except for either a low neutrophil count (neutropenia) or a low platelet count (thrombocytopenia)
Measured: 2 years
Secondary outcomes (8)
Overall response rate (ORR)
ORR is defined as the proportion of patients achieving CR + CRh + CRi + Partial Remission (PR) + morphologic leukemia-free state [MLFS] per ELN 2022.
PR is defined as all hematologic criteria of CR; decrease of bone marrow blast percentage to 5% to 25%; and decrease of pretreatment bone marrow blast percentage by at least 50%. MLFS is defined as bone marrow blasts < 5%, absence of circulating blasts, and no hematologic recovery.
Measured: 2 years
Overall survival
OS is defined as the time from registration to death from any cause or last known follow-up.
Measured: 2 years
Duration of response (DoR)
DoR is defined as the time between the first achieved response (CR, CRh, CRi, PR, MLFS) and the earliest evidence of relapse (blasts ≥ 5%) per ELN 2022 or death or date of last follow up.
Measured: 2 years
Time to hematologic improvement (hemoglobin)
Improvement of Hgb is defined as the time it takes for an individual's hemoglobin level to increase by ≥ 1g/dL from Cycle 1 Day 1 without transfusion
Measured: 2 years
Time to hematologic improvement (platelet count)
Improvement of PLT is defined as the time it takes for an individual's platelet count to increase by ≥ 50 x 10^9/L from Cycle 1 Day 1 without transfusion
Measured: 2 years
Time to hematologic improvement (neutrophil count)
Improvement of ANC is defined as the time it takes for an individual's neutrophil count to increase to ≥ 1,500 cells/µL for 3 days.
Measured: 2 years
Rate of transfusion independence
The rate of transfusion independence is defined as the absence of red blood cell (RBC) transfusions for ≥ 8 weeks following the start of treatment
Measured: 2 years
Rate of allogeneic transplant
The rate of allogeneic transplant is defined as the proportion of patients who undergo allogeneic transplant following response to olutasidenib.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2026
- Study startNov 2026 (estimated)
- Primary completionJun 2028 (estimated)
- Study completionJun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for IDH1 MutationStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Timothy Pardee
- Sponsor type
- University, hospital or other
- Collaborators
- Atrium Health Wake Forest Baptist, Rigel Pharmaceuticals
- Responsible party
- Sponsor-investigator, Timothy Pardee, Hoosier Cancer Research Network
- Investigators
- Timothy Pardee, MD, PhD, Principal investigator, Atrium Health Wake Forest Baptist
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07471841
- Sponsor's protocol ID
- HCRN-AML24-687
- Status last verified
- Nov 2026
- First submitted
- 10 Mar 2026
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Single Arm Phase 2 Study of Olutasidenib in Relapsed IDH1 Mutated AML Patients Who Have Previously Received Venetoclax
- ClinicalTrials.gov ID
- NCT07471841
- Acronym / sponsor ID
- HCRN-AML24-687
- Conditions
- IDH1 Mutation, Relapsed / Refractory AML
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Timothy PardeeUniversity, hospital or other
- Collaborators
- Atrium Health Wake Forest Baptist, Rigel Pharmaceuticals
- Study start
- Nov 2026 (estimated)
- Primary completion
- Jun 2028 (estimated)
- Study completion
- Jun 2029 (estimated)
- First posted
- 13 Mar 2026 (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 prospective, single-arm phase 2 pilot study to assess the response rate of IDH1 mutated relapsed/refractory acute myeloid leukemia (AML) patients who receive olutasidenib after progressing on venetoclax based regimens. Each cycle will last for 28 days. Patients will receive olutasidenib 150 mg orally twice daily Day 1 through Day 28. After 3 cycles of olutasidenib, azacitidine 75 mg/m2 given on Day 1 through Day 7 may be added at the discretion of the treating investigator if the patient has not achieved a complete remission. Subjects with at least a PR after 6 cycles of treatment will continue treatment as previously described. Subjects without at least a partial response (PR) after 6 cycles of treatment will move to long term follow up.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Olutasidenib and Azacitidine | Experimental | Drug: Olutasidenib, Drug: Azacitidine (AZA)Each cycle will last for 28 days. Patients will receive olutasidenib 150 mg orally twice daily Day 1 through Day 28. After 3 cycles of olutasidenib, azacitidine 75 mg/m2 given on Day 1 through Day 7 may be added at the discretion of the treating investigator if the patient has not achieved a complete remission or for whom loss of response is suspected. Subjects with at least a PR after 6 cycles of treatment will continue treatment.. Subjects without at least a PR after 6 cycles of treatment will move to long term follow up. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Written informed consent and HIPAA authorization for release of personal health information prior to registration. NOTE: HIPAA authorization may be included in the informed consent or obtained separately.
- Age ≥ 18 years at the time of consent.
- ECOG Performance Status of ≤ 2 within 28 days prior to registration.
- Must have histologically or cytologically documented relapsed and/or refractory Acute Myeloid Leukemia (Refractory is defined as failure to achieve a CR after induction chemotherapy or a minimum of two cycles of HMA plus venetoclax)
- Acute Myeloid Leukemia with a documented IDH1 mutation.
Key exclusion criteria (10 listed)
- Previous exposure to ivosidenib or any IDH1 inhibitor.
- Active infection requiring IV systemic therapy. NOTE: Subjects receiving prophylactic antibiotics (e.g., to prevent a urinary tract infection or chronic obstructive pulmonary disease exacerbation) are eligible for the study.
- Known CNS involvement with AML.
- Previous allogeneic stem cell transplant within 60 days prior to registration.
- Treatment with any investigational drug within 21 days or 2 half-life's prior to registration.
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 |
|---|---|---|
| OlutasidenibOlutasidenib 150 mg orally twice a day | Drug | Olutasidenib and Azacitidine |
| Azacitidine (AZA)Azacitidine 75 mg/m2 subcutaneously or IV (over 10-40 minutes) | Drug | Olutasidenib and Azacitidine |
Primary outcome
Composite complete remission rate
Time frame: 2 years
Composite complete remission (CRc) rate is defined as patients that meet the criteria for CR + CRh + CRi per the modified European LeukemiaNet (mELN) 2022. * Complete Remission (CR) is defined as absence of leukemia cells in the bone marrow (< 5% blasts), normal blood counts (absolute neutrophil count ≥ 1000/μL and platelet count ≥ 100,000/μL), and the absence of circulating blasts, and extramedullary disease * Complete Remission with Hematologic recovery (CRh) is defined as meeting all criteria for CR as Bone marrow myeloblasts < 5%; absence of circulating blasts; absence of extramedullary disease; both ANC ≥ 0.5x109/L (500/µL) and platelet count ≥ 50×109/L (50 000/µL) * Complete Remission with Incomplete hematologic recovery (CRi) is defined as meeting all criteria for Complete Remission (CR) except for either a low neutrophil count (neutropenia) or a low platelet count (thrombocytopenia)
8 secondary outcomes
Overall response rate (ORR)
Time frame: 2 years
Overall survival
Time frame: 2 years
Duration of response (DoR)
Time frame: 2 years
Time to hematologic improvement (hemoglobin)
Time frame: 2 years
Time to hematologic improvement (platelet count)
Time frame: 2 years
Time to hematologic improvement (neutrophil count)
Time frame: 2 years
Rate of transfusion independence
Time frame: 2 years
Rate of allogeneic transplant
Time frame: 2 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 4 Nov 2026.
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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.
Timothy Pardee, MD, PhD principal investigator
Atrium Health Wake Forest Baptist
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 Olutasidenib and Azacitidine (AZA) in people with IDH1 Mutation and Relapsed / Refractory AML, run by Timothy Pardee at 1 site 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 19 inclusion and 10 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 4 Nov 2026. 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 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 Timothy Pardee (MD, PhD, Timothy.Pardee@wfusm.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Composite complete remission rate (time frame: 2 years). 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 4 Nov 2026.