Myelodysplastic SyndromesPhase 2NCT06566742
A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia.
A Phase 2 study testing Olutasidenib in people with Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia, run by M.D. Anderson Cancer Center at 2 sites in United States.
Trial summary
To learn if olutasidenib can help to control CCUS, MDS, and/or CMML. The safety of the drug will also be studied. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Clonal Cytopenia of Undetermined Significance
- What is being tested?
- Olutasidenib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesUnited States
- Study dates
- 10 Dec 2024to 31 Aug 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
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- To be considered eligible to participate in this study, a patient must meet ALL inclusion criteria as follows:
- Pathologically proven CCUS or lower-risk MDS/CMML.
- CCUS is defined as the presence of cytopenia (absolute neutrophil count < 1.8 x 10^9/L, hemoglobin < 13 g/dL in males or < 12 g/dL in females, and/or platelets < 150 x 10^9/L) for at least 30 days that are otherwise unexplained and with no diagnostic hematopathologic features of myeloid neoplasms. Patients with known Duffy-null phenotype must have absolute neutrophil counts less than their lower limit of normal.
- Lower-risk MDS/CMML includes patients with International Prognostic Scoring System (IPSS) low- or intermediate-1-risk disease and Revised IPSS (IPSS-R) score ≤ 3.5 and Molecular IPSS (IPSS-M) very low-, low-, or moderate low-risk categories.
- Patients must have a documented IDH1 mutation with variant allele frequency (VAF) ≥ 0.02.
- Patients ≥ 18 years old.
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- 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 patients with Gilbert Syndrome.
- Aspartate transaminase (AST), alanine transaminase (ALT), and alkaline phosphatase ≤ 3 times ULN.
- Acceptable renal function with serum creatinine ≤ 1.5 times ULN or calculated creatinine clearance ≥ 50 mL/min (as assessed by Cockcroft-Gault, Modification of Diet in Renal Disease Formula [MDRD], or Chronic Kidney Disease Epidemiology [CKD-Epi] validated measures).
- Negative serum or urine pregnancy test if female of childbearing potential.
- 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. Appropriate highly effective method(s) of contraception include oral or injectable hormonal birth control, intrauterine device (IUD), and double barrier methods (for example a condom in combination with a spermicide).
- Agreement for male patients not to donate sperm and for female patients 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 signed informed consent prior to beginning study and undergoing procedures.
Who cannot join
- To be eligible for entry into the study, the patient must NOT meet any of the exclusion criteria listed below:
- Patients unable to swallow oral medications, or patients with gastrointestinal conditions (e.g., malabsorption, resection, etc.) deemed by the Investigator to jeopardize intestinal absorption.
- Patients with any concurrent uncontrolled clinically significant medical condition, including life-threatening severe infection or psychiatric illness, which could place the patient at unacceptable risk of study treatment.
- Known active hepatitis B (hepatitis B virus [HBV]) or hepatitis C (hepatitis C virus [HCV]) or HIV infection.
- Pregnant or nursing women or women of childbearing potential not using highly effective contraception; male patients not using highly effective contraception as defined in the inclusion criteria.
- Subject with white blood cell count > 25 x10^9/L.
- Note: hydroxyurea use is permitted to meet this criterion with no washout required.
- Unwillingness or inability to comply with procedures either required in this protocol or considered standard of care.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
Kelly Chien, MD, 713-745-7584, kchien@mdanderson.org
Kelly Chien, MD
- Recruiting
University of Miami
Miami, Florida, 33136
Sangeetha Venugopal, MD, 305-243-6718, svenugopal@miami.edu
Sangeetha Venugopal, MD
About this study
To learn if olutasidenib can help to control CCUS, MDS, and/or CMML. The safety of the drug will also be studied.
Read the detailed description
Primary Objectives - To determine the response rate of olutasidenib monotherapy in patients with IDH1-mutated CCUS or lower-risk MDS/CMML
Secondary Objectives
- To evaluate the rates of transfusion independence, defined as the absence of transfusions over a period of at least 8 weeks
- To ascertain the safety and tolerability of olutasidenib monotherapy in these participants populations
- To determine survival and rates of leukemia transformation
- To analyze reduction in IDH1 clone size
Exploratory Objectives
- 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.
OUTLINE:
Patients receive olutasidenib orally (PO) twice daily (BID) on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients with CCUS receive up to 18 months of olutasidenib. Patients with lower-risk MDS/CMML can receive olutasidenib until disease progression or unacceptable toxicity. Additionally, patients undergo blood sample collection and bone marrow aspiration and biopsy on study.
After completion of study treatment, patients are followed up every 3 months for up to 3 years.
- Conditions
- Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Clonal Cytopenia of Undetermined Significance
- Being tested
- Olutasidenib
- Medical subject headings
- Myelodysplastic Syndromes, 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 | 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. 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 posted22 Aug 2024
- Study start10 Dec 2024
- Primary completion31 Aug 2027 (estimated)
- Study completion31 Aug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies 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
- 15 participants (estimated)
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
- NCT06566742
- Sponsor's protocol ID
- 2024-0509
- Other IDs
- NCI-2024-07045
- Status last verified
- Sep 2026
- First submitted
- 20 Aug 2024
- Last updated
- 11 Sep 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 11 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia.
- ClinicalTrials.gov ID
- NCT06566742
- Acronym / sponsor ID
- 2024-0509
- Conditions
- Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Clonal Cytopenia of Undetermined Significance
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Rigel Pharmaceuticals
- Study start
- 10 Dec 2024 (actual)
- Primary completion
- 31 Aug 2027 (estimated)
- Study completion
- 31 Aug 2029 (estimated)
- First posted
- 22 Aug 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 can help to control CCUS, MDS, and/or CMML. The safety of the drug will also be studied.
Detailed description
Primary Objectives - To determine the response rate of olutasidenib monotherapy in patients with IDH1-mutated CCUS or lower-risk MDS/CMML
Secondary Objectives
- To evaluate the rates of transfusion independence, defined as the absence of transfusions over a period of at least 8 weeks
- To ascertain the safety and tolerability of olutasidenib monotherapy in these participants populations
- To determine survival and rates of leukemia transformation
- To analyze reduction in IDH1 clone size
Exploratory Objectives
- 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.
OUTLINE:
Patients receive olutasidenib orally (PO) twice daily (BID) on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients with CCUS receive up to 18 months of olutasidenib. Patients with lower-risk MDS/CMML can receive olutasidenib until disease progression or unacceptable toxicity. Additionally, patients undergo blood sample collection and bone marrow aspiration and biopsy on study.
After completion of study treatment, patients are followed up every 3 months for up to 3 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Olutasidenib | 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- To be considered eligible to participate in this study, a patient must meet ALL inclusion criteria as follows:
- Pathologically proven CCUS or lower-risk MDS/CMML.
- Patients must have a documented IDH1 mutation with variant allele frequency (VAF) ≥ 0.02.
- Patients ≥ 18 years old.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 (Appendix A)
Key exclusion criteria (8 listed)
- To be eligible for entry into the study, the patient must NOT meet any of the exclusion criteria listed below:
- Patients unable to swallow oral medications, or patients with gastrointestinal conditions (e.g., malabsorption, resection, etc.) deemed by the Investigator to jeopardize intestinal absorption.
- Patients with any concurrent uncontrolled clinically significant medical condition, including life-threatening severe infection or psychiatric illness, which could place the patient at unacceptable risk of study treatment.
- Known active hepatitis B (hepatitis B virus [HBV]) or hepatitis C (hepatitis C virus [HCV]) or HIV infection.
- Pregnant or nursing women or women of childbearing potential not using highly effective contraception; male patients not using highly effective contraception as defined in the inclusion criteria.
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 |
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
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 11 Sep 2026.
- MD Anderson Cancer CenterLink provided by the sponsor.
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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.
Kelly Chien, MD principal investigator
M.D. Anderson Cancer Center
2 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 Olutasidenib in people with Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia, run by M.D. Anderson Cancer Center at 2 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 15 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 11 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 Kelly Chien, MD ((713) 745-7584, kchien@mdanderson.org), or a site near the patient. 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 11 Sep 2026.