Clonal Cytopenia of Undetermined SignificancePhase 2NCT06240754
Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2A Decentralized Trial
A Phase 2 study testing Enasidenib in people with Clonal Cytopenia of Undetermined Significance and CCUS Clonal Cytopenia of Undetermined Significance, run by Washington University School of Medicine at 1 site in United States.
Trial summary
Study researchers think that a drug called enasidenib may help people with clonal cytopenia of undetermined significance (CCUS) because the drug blocks the mutated IDH2 protein, which may improve blood cell counts. The purpose of this study is to find out whether enasidenib is a safe and effective treatment for CCUS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Clonal Cytopenia of Undetermined Significance, CCUS Clonal Cytopenia of Undetermined Significance
- What is being tested?
- Enasidenib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Oct 2024to 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
- Unexplained cytopenia for at least 6 months. Cytopenia is defined as the presence of ≥1 blood count indexes below the following thresholds:
- Hgb <10 g/dL
- ANC <1.8 × 109/L
- Platelets <100 × 109/L
- IDH2 gene mutation (R140 or R172), performed locally, at a frequency ≥ 2%.
- At least 18 years of age.
Show all 13Show fewer
- ECOG performance status 0-2
- Adequate organ function as defined below:
- AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN
- Serum total bilirubin < 1.5 x IULN (un upper limit of bilirubin 5 mg/dL is acceptable if it can be attributed to Gilbert's syndrome or erythropoiesis)
- Creatinine clearance > 50 mL/min by Cockcroft-Gault glomerular filtration rate estimation or serum creatinine ≤ 2 x IULN
- The effects of enasidenib on the developing human fetus are unknown. For this reason, women of childbearing potential and men must agree to use adequate contraception prior to study entry, for the duration of study participation, and for 24 months after the last dose of enasidenib. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of the study, and for 4 months after the last dose of enasidenib.
- Ability to understand and willingness to sign an IRB approved written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants.
Who cannot join
- Indication of hematologic disease by bone marrow biopsy within 6 months of study entry.
- Evidence of disease progression from time of bone marrow biopsy to enrollment based on investigator review of symptoms and complete blood counts
- Active malignancy (defined as > 1 cm disease on most recent CT scan in the past 6 months).
- Currently receiving therapy for solid tumor malignancy or received within the last 6 months.
- Currently receiving any other investigational agents.
- Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally.
Show all 10Show fewer
- A history of allergic reactions attributed to compounds of similar chemical or biologic composition to enasidenib or other agents used in the study.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
- Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 72 hours of study entry.
- Positive direct Coombs test.
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
Washington University School of Medicine
St Louis, Missouri, 63110
Giulia Petrone, M.D., 314-362-6826, gpetrone@wustl.edu
Giulia Petrone, M.D.
About this study
Official title: A Pilot Study of Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2: A Decentralized Trial
Study researchers think that a drug called enasidenib may help people with clonal cytopenia of undetermined significance (CCUS) because the drug blocks the mutated IDH2 protein, which may improve blood cell counts. The purpose of this study is to find out whether enasidenib is a safe and effective treatment for CCUS.
- Conditions
- Clonal Cytopenia of Undetermined Significance, CCUS Clonal Cytopenia of Undetermined Significance
- Being tested
- Enasidenib
- Keywords
- Clonal Cytopenia of Undetermined Significance, CCUS, IDH2, Enasidenib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EnasidenibDrug
Provided by BMS.
| Study group | Role | What they receive |
|---|---|---|
| Enasidenib | Experimental | Participants will receive enasidenib 100 mg daily for 18 cycles (each cycle is 28 days).
Participants will continue treatment with enasidenib until confirmed progression to AML or MDS, development of unacceptable toxicity, or suspicion of disease progression, provided the patient is deriving clinical benefit, which will be determined at the discretion of the principal investigator. Receives: Drug: Enasidenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Best hematologic response
Hematologic response to enasidenib will be evaluated according to a modified version of the IWG 2006 Criteria for Hematologic Improvement for patients with MDS on clinical trials
Measured: Up to 18 cycles (each cycle is 28 days) of treatment (up to approximately 17 months)
Secondary outcomes (3)
Toxicity as measured by the number of adverse events experienced by participant
Measured by CTCAE v 5.0
Measured: From start of treatment through 30 days after the last day of treatment (up to approximately 18 months)
Change in mutant IDH2 variant allele fraction
The IDH2 variant allele fraction reflects the clonal dominance in the blood. Blood samples will be drawn at various time points throughout the study to determine the IDH2 variant allele fraction. The lower the IDH2 variant allele fraction the better the outcome.
Measured: Baseline, day 1 of cycles 3/6/9/12/15 (each cycle is 28 days), and end of treatment (up to approximately 17 months)
Duration of hematologic improvement
Measured: From start of treatment through completion of treatment (estimated to be 17 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2024
- Study start10 Oct 2024
- Primary completion31 May 2028 (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 Clonal Cytopenia of Undetermined SignificanceStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe 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
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Bristol-Myers Squibb, Damon Runyon Cancer Research Foundation
- Responsible party
- Sponsor
- Investigators
- Giulia Petrone, M.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06240754
- Sponsor's protocol ID
- 202405007
- Status last verified
- Aug 2026
- First submitted
- 16 Jan 2024
- Last updated
- 10 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant data that underlie the results reported in this article after deidentification (including text, tables, figures and appendices) will be available.
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2: A Decentralized Trial
- ClinicalTrials.gov ID
- NCT06240754
- Acronym / sponsor ID
- 202405007
- Conditions
- Clonal Cytopenia of Undetermined Significance, CCUS Clonal Cytopenia of Undetermined Significance
- Keywords
- Clonal Cytopenia of Undetermined Significance, CCUS, IDH2, Enasidenib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Collaborators
- Bristol-Myers Squibb, Damon Runyon Cancer Research Foundation
- Study start
- 10 Oct 2024 (actual)
- Primary completion
- 31 May 2028 (estimated)
- Study completion
- 30 Jun 2029 (estimated)
- First posted
- 5 Feb 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.
Study researchers think that a drug called enasidenib may help people with clonal cytopenia of undetermined significance (CCUS) because the drug blocks the mutated IDH2 protein, which may improve blood cell counts. The purpose of this study is to find out whether enasidenib is a safe and effective treatment for CCUS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Enasidenib | Experimental | Drug: EnasidenibParticipants will receive enasidenib 100 mg daily for 18 cycles (each cycle is 28 days). Participants will continue treatment with enasidenib until confirmed progression to AML or MDS, development of unacceptable toxicity, or suspicion of disease progression, provided the patient is deriving clinical benefit, which will be determined at the discretion of the principal investigator. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Unexplained cytopenia for at least 6 months. Cytopenia is defined as the presence of ≥1 blood count indexes below the following thresholds:
- IDH2 gene mutation (R140 or R172), performed locally, at a frequency ≥ 2%.
- At least 18 years of age.
- ECOG performance status 0-2
- Adequate organ function as defined below:
Key exclusion criteria (10 listed)
- Indication of hematologic disease by bone marrow biopsy within 6 months of study entry.
- Active malignancy (defined as > 1 cm disease on most recent CT scan in the past 6 months).
- Currently receiving therapy for solid tumor malignancy or received within the last 6 months.
- Currently receiving any other investigational agents.
- Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally.
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 |
|---|---|---|
| EnasidenibProvided by BMS. | Drug | Enasidenib |
Primary outcome
Best hematologic response
Time frame: Up to 18 cycles (each cycle is 28 days) of treatment (up to approximately 17 months)
Hematologic response to enasidenib will be evaluated according to a modified version of the IWG 2006 Criteria for Hematologic Improvement for patients with MDS on clinical trials
3 secondary outcomes
Toxicity as measured by the number of adverse events experienced by participant
Time frame: From start of treatment through 30 days after the last day of treatment (up to approximately 18 months)
Change in mutant IDH2 variant allele fraction
Time frame: Baseline, day 1 of cycles 3/6/9/12/15 (each cycle is 28 days), and end of treatment (up to approximately 17 months)
Duration of hematologic improvement
Time frame: From start of treatment through completion of treatment (estimated to be 17 months)
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 10 Aug 2026.
- Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Individual participant data that underlie the results reported in this article after deidentification (including text, tables, figures and appendices) will be available.
Related trials for Clonal Cytopenia of Undetermined Significance
RecruitingObservational study
Clonal Hematopoiesis and Therapy-Emergent Myeloid Neoplasms in Patients With Cancers, CHANCES Study
Recurrent Fallopian Tube Carcinoma, Recurrent Ovarian Carcinoma
Not yet recruitingPhase 2
Modulation of Stem Cell Differentiation in Individuals With High Risk Clonal Haematopoiesis
Clonal Cytopenia of Uncertain Significance, CCUS Clonal Cytopenia of Undetermined Significance
RecruitingPhase 1
Safety and Tolerability of REGN17235 in Adult Participants With Clonal Cytopenia of Undetermined Significance and Low-Risk Myelodysplastic Syndrome With SF3B1 Mutation
Clonal Cytopenia of Undetermined Significance (CCUS), Low-Risk Myelodysplastic Syndrome (LR-MDS)
RecruitingPhase 2
Ascorbic Acid and Chemotherapy for the Treatment of Relapsed or Refractory Lymphoma, CCUS, and Chronic Myelomonocytic Leukemia
Clonal Cytopenia of Undetermined Significance, High Grade B-Cell Lymphoma With MYC and BCL2 or BCL6 Rearrangements
RecruitingObservational study
CHIP/CCUS Natural History Protocol
Clonal Hematopoiesis of Indeterminate Potential, Clonal Cytopenia of Undetermined Significance
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.
Giulia Petrone, M.D. principal investigator
Washington University School of Medicine
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 Enasidenib in people with Clonal Cytopenia of Undetermined Significance and CCUS Clonal Cytopenia of Undetermined Significance, run by Washington University School of Medicine 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 13 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 10 Aug 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 Giulia Petrone, M.D. (314-362-6826, gpetrone@wustl.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Best hematologic response (time frame: Up to 18 cycles (each cycle is 28 days) of treatment (up to approximately 17 months)). 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 10 Aug 2026.