Chronic Myelomonocytic LeukemiaPhase 2NCT06597734
A Phase 2 Study Evaluating Olutasidenib in Combination With Hypomethylating Agents in Patients With IDH1-mutated Higher-risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Advanced Myeloproliferative Neoplasm
A Phase 2 study testing Olutasidenib in people with Chronic Myelomonocytic Leukemia and Advanced Myeloproliferative Neoplasms, run by M.D. Anderson Cancer Center.
Trial summary
To learn if olutasidenib, when combined with a drug called a hypomethylating agent (HMA) can help to control MDS, CMML, and/or MPN. The safety of the drug combination will also be studied. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myelomonocytic Leukemia, Advanced Myeloproliferative Neoplasms, IDH1-mutated Higher-Risk Myelodysplastic Syndromes
- What is being tested?
- Olutasidenib
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 28 Jan 2025to 11 Jun 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Pathologically proven higher-risk MDS/CMML or advanced MPN
- MDS participants must have International Prognostic Scoring System (IPSS) intermediate-2- or high-risk disease or Revised IPSS (IPSS-R) score >= 3.5 or Molecular IPSS (IPSS-M) moderate high-, high-, or very high-risk disease or bone marrow blast percentage.
- CMML patients must have CPSS-Mol int-1, int-2, or high-ris disease (Elena C et al, Blood 2016)
- Advanced MPN is defined as bone marrow blast percentage >=/=10%.
- Participants on the treatment-naive arm must not have received a prior HMA. Agents such as growth factors (e.g. erythropoietin stimulating agents, luspatercept, eltrombopag, granulocyte colony stimulating factors), cyclosporine, and/or hydroxyurea are allowed.
- Patients on the previously-treated arm must have received a prior HMA and/or ivosidenib. Prior stem cell transplantation in allowed>
Show all 16Show fewer
- Participants must have a documented IDH1 mutation
- Patients with previously-treated MDS must be ineligible to receive treatment with ivosidenib or have progressed on treatment with ivosidenib.
- Participants >=/= 18 years old
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 (Appendix A)
- Acceptable liver function
- Bilirubin </= 2 times upper limit of normal (ULN) or <;/= 3 times ULN in participants with Gilbert Syndrome
- Aspartate transaminase (AST), alanine transaminase (ALT), and alkaline phosphatase </= 3 times ULN
- Acceptable renal function calculated creatinine clearance >/= 50 mL/min (as assessed by Cockcroft-Gault, MDRD, or CKD-Epi validated measures)
- Negative serum or urine pregnancy test if female of childbearing potential c. For fertile men and women, agreement to use highly effective contraceptive methods for the duration of study participation and 90 days after the last dose of study medication d. Agreement for male participants not to donate sperm and for female participants of childbearing potential not to donate ova during the study and for 90 days after the final dose of study drug
- Ability and willingness to sign informed consent prior to beginning study and undergoing procedures
Who cannot join
- Participants unable to swallow oral medications, or participants with gastrointestinal conditions (e.g., malabsorption, resection, etc.) deemed by the Investigator to jeopardize intestinal absorption
- Participants with any concurrent uncontrolled clinically significant medical condition, including life-threatening severe infection or psychiatric illness, which could place the participant at unacceptable risk of study treatment
- Patients must have discontinued prior chemotherapy at least 1 week prior to the start of study treatment. Patients with MPN must be off JAK inhibitors at the start of study treatment
- Patients with active graft-versus-host-disease (GVHD) status post stem cell transplantation, including active chronic GVHD requiring topical therapy. Patients must have discontinued calcineurin inhibitors at least 4 weeks prior to the start of study treatment
- Known active hepatitis B (HBV) or hepatitis C (HCV) or HIV infection
- Pregnant or nursing women or women of childbearing potential not using highly effective contraception; male participants not using highly effective contraception
- Participants with white blood cell count \> 25 x109/L Note: hydroxyurea use is permitted to meet this criterion
- Unwillingness or inability to comply with procedures either required in this protocol or considered standard of care
About this study
To learn if olutasidenib, when combined with a drug called a hypomethylating agent (HMA) can help to control MDS, CMML, and/or MPN. The safety of the drug combination will also be studied.
Read the detailed description
Primary Objectives To determine the overall response rate of olutasidenib in combination with investigator's choice of HMA in patients with IDH1-mutated higher-risk MDS/CMML or advanced MPN Secondary Objectives
The secondary objectives of this study are:
- To evaluate the rates of complete remission (CR) and median duration of CR
- To ascertain the safety and tolerability of olutasidenib with HMA in this participant population
- To determine survival including overall survival (OS), progression-free survival (PFS), and duration of response (DOR) To analyze reduction in IDH1 clone size Exploratory Objectives
- To examine overall response rate of patients previously exposed to venetoclax
- To investigate global gene expression profiles, DNA methylation profiles, and other potential prognostic markers to explore predictors of antitumor activity and/or resistance to treatment
- Conditions
- Chronic Myelomonocytic Leukemia, Advanced Myeloproliferative Neoplasms, IDH1-mutated Higher-Risk Myelodysplastic Syndromes
- Being tested
- Olutasidenib
- Medical subject headings
- Leukemia, Myelomonocytic, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OlutasidenibDrug
Given by PO
| Study group | Role | What they receive |
|---|---|---|
| Olutasidenib+Azacitidine-IV or SubQ | Experimental | Participants will take capsules of olutasidenib 2 times each day while you are on study. Each dose should be taken about 12 hours apart at least 1 hour before or 2 hours after a meal. Azacitidine IV or Sub Q for 7 days every 28 days. Receives: Drug: Olutasidenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and adverse events (AEs)
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Measured: Through study completion; an average of 1 year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start28 Jan 2025
- Primary completion11 Jun 2026
- Study completion11 Jun 2026
Why it stopped: PI request
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myelomonocytic LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Rigel Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Kelly Chien, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06597734
- Sponsor's protocol ID
- 2024-0515
- Other IDs
- NCI-2024-07758
- Status last verified
- Sep 2026
- First submitted
- 12 Sep 2024
- Last updated
- 5 Oct 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 5 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study Evaluating Olutasidenib in Combination With Hypomethylating Agents in Patients With IDH1-mutated Higher-risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Advanced Myeloproliferative Neoplasm
- ClinicalTrials.gov ID
- NCT06597734
- Acronym / sponsor ID
- 2024-0515
- Conditions
- Chronic Myelomonocytic Leukemia, Advanced Myeloproliferative Neoplasms, IDH1-mutated Higher-Risk Myelodysplastic Syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: PI request
- Enrolment
- 0 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Rigel Pharmaceuticals
- Study start
- 28 Jan 2025 (actual)
- Primary completion
- 11 Jun 2026 (actual)
- Study completion
- 11 Jun 2026 (actual)
- First posted
- 19 Sep 2024 (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.
To learn if olutasidenib, when combined with a drug called a hypomethylating agent (HMA) can help to control MDS, CMML, and/or MPN. The safety of the drug combination will also be studied.
Detailed description
Primary Objectives To determine the overall response rate of olutasidenib in combination with investigator's choice of HMA in patients with IDH1-mutated higher-risk MDS/CMML or advanced MPN Secondary Objectives
The secondary objectives of this study are:
- To evaluate the rates of complete remission (CR) and median duration of CR
- To ascertain the safety and tolerability of olutasidenib with HMA in this participant population
- To determine survival including overall survival (OS), progression-free survival (PFS), and duration of response (DOR) To analyze reduction in IDH1 clone size Exploratory Objectives
- To examine overall response rate of patients previously exposed to venetoclax
- To investigate global gene expression profiles, DNA methylation profiles, and other potential prognostic markers to explore predictors of antitumor activity and/or resistance to treatment
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Olutasidenib+Azacitidine-IV or SubQ | Experimental | Drug: OlutasidenibParticipants will take capsules of olutasidenib 2 times each day while you are on study. Each dose should be taken about 12 hours apart at least 1 hour before or 2 hours after a meal. Azacitidine IV or Sub Q for 7 days every 28 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Pathologically proven higher-risk MDS/CMML or advanced MPN
- Participants must have a documented IDH1 mutation
- Patients with previously-treated MDS must be ineligible to receive treatment with ivosidenib or have progressed on treatment with ivosidenib.
- Participants >=/= 18 years old
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 (Appendix A)
Key exclusion criteria (8 listed)
- Participants unable to swallow oral medications, or participants with gastrointestinal conditions (e.g., malabsorption, resection, etc.) deemed by the Investigator to jeopardize intestinal absorption
- Participants with any concurrent uncontrolled clinically significant medical condition, including life-threatening severe infection or psychiatric illness, which could place the participant at unacceptable risk of study treatment
- Patients must have discontinued prior chemotherapy at least 1 week prior to the start of study treatment. Patients with MPN must be off JAK inhibitors at the start of study treatment
- Patients with active graft-versus-host-disease (GVHD) status post stem cell transplantation, including active chronic GVHD requiring topical therapy. Patients must have discontinued calcineurin inhibitors at least 4 weeks prior to the start of study treatment
- Known active hepatitis B (HBV) or hepatitis C (HCV) or HIV infection
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 |
|---|---|---|
| OlutasidenibGiven by PO | Drug | Olutasidenib+Azacitidine-IV or SubQ |
Primary outcome
Safety and adverse events (AEs)
Time frame: Through study completion; an average of 1 year.
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2026.
- MD Anderson Cancer CenterLink provided by the sponsor.
Related trials for Chronic Myelomonocytic Leukemia
RecruitingPhase 1
Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Hematologic Malignancies
Malignancy, Hematologic, Neoplasms, Hematologic
Active, not recruitingPhase 2
Cobimetinib in Newly Diagnosed or HMA-treated CMML Patients With RAS Pathway Mutations
Chronic Myelomonocytic Leukemia (CMML)
Active, not recruitingObservational study
Infection Prophylaxis and Management in Treating Cytomegalovirus (CMV) Infection in Patients With Hematologic Malignancies Previously Treated With Donor Stem Cell Transplant
Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia
RecruitingPhase 1
Therapeutic RSK1 Targeting in Myeloid Malignancies
Myeloid Malignancy
RecruitingPhase 1
Olutasidenib for the Treatment of Patients With IDH1 Mutated AML, MDS or CMML After Donor Hematopoietic Cell Transplant
Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia
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. This study is not recruiting at the moment.
Kelly Chien, MD principal investigator
M.D. Anderson Cancer Center
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Olutasidenib in people with Chronic Myelomonocytic Leukemia and Advanced Myeloproliferative Neoplasms, run by M.D. Anderson Cancer Center.
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 16 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: withdrawn, as of the sponsor's last update on 5 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and adverse events (AEs) (time frame: Through study completion; an average of 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 5 Oct 2026.