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LeukemiaPhase 1/2NCT05282459

Enasidenib in MDS &Non-proliferative Chronic Myelomonocytic Leukemia w/o IDH2 Mutation

A Phase 1/2 study testing Enasidenib mesylat dose escalation in people with Leukemia and Leukemia, Myeloid, run by Tian Yi Zhang at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a phase 1b/2, open-label, single arm study to evaluate if enasidenib is safe and effective in improving anemia and decreasing transfusion needs in subjects diagnosed with lower risk myelodysplastic syndrome (MDS) or nonproliferative chronic myelomonocytic leukemia (CMML) without a mutation in isocitrate… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Leukemia, Leukemia, Myeloid, Monocytic Leukemia
What is being tested?
Enasidenib mesylat dose escalation
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
17 participants
Where
1 siteUnited States
Study dates
12 Jan 2022to 3 Mar 2025
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • Documented diagnosis of
  • MDS according to WHO/FAB classification that meets IRSS-R classification of low or intermediate risk disease; and a diagnosed as denovo or secondary MDS (MDS-RS eligible if refractory to or declined luspatercept therapy) OR
  • Dysplastic (nonproliferative) CMML with WBC < 13.0/microL)
  • No disease-modifying therapy (HMA, hydrea) within 2 months of starting study
  • Age ≥ 18 years of age
  • ECOG ≤ 3
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  • Negative for IDH2 mutation by NGS or multiplex PCR (SNaPshot)
  • Has symptomatic anemia defined as hemoglobin < 10.5 g/dL with any of the following.
  • Tachypnea
  • Shortness of breath
  • Fatigue
  • Malaise
  • Worsening of cardiovascular function
  • Asthenia
  • Dyspnea on exertion
  • Angina
  • Other subject symptoms the subject reports as being associated with being anemic.
  • Stated willingness to comply with all study procedures and availability for the duration of the study
  • Ability to take oral medication and be willing to adhere to the medication regimen.
  • Females of reproductive potential need to either commit to true abstinence from heterosexual contact or agree to use, and be able to comply with highly effective contraception without interruption, 28 days prior to starting enasidenib, during the study therapy, and for 30 days after last dose of enasidenib
  • For males of reproductive potential: agreement to use of condoms
  • Adequate organ function defined as:
  • Hepatic function: total bilirubin <1.5 x ULN (unless attributable to Gilbert's disease), AST or ALT < 3x ULN
  • Renal function: creatinine clearance > 30 mL/minute, calculated by Cockcroft-Gault formula
  • Ability to understand and the willingness to sign the IRB approved informed consent document.
  • Women of childbearing potential must have negative urine or serum pregnancy test

Who cannot join

  • Use of concurrent other erythropoietic agents (including epoetin, darbepoetin), G-CSF within 30 days of study enrollment
  • Less than 3 months of life expectancy
  • Significant cardiac disease (NYHA Class IV congestive heart failure, or unstable angina or myocardial infarction within the last 6 months
  • Harbor IDH2 somatic mutations by NGS or PCR
  • Pregnant or breast feeding
  • Any uncontrolled bacterial, fungal, viral or other infection.
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  • No known HIV+ or active hepatitis B or C infection, defined as positive viral load for HBV or HCV or a positive surface antigen (HBsAg) test for hepatitis B.
  • Have other causes of anemia: deficiencies in iron, B12, folate; nutritional deficiencies related to gastric surgery, anorexia nervosa, excessive zinc supplementation; gastrointestinal bleed. If nutritional deficiencies can be corrected, potential subject can be rescreened and enrolled if nutritionally replete and still meets eligibility criteria.
  • Any other medical history, including laboratory results, deemed by the Principal Investigator likely to interfere with their participation in the study, or to interfere with the interpretation of the results
  • Pregnant or breast feeding

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Stanford Cancer Institute

    Palo Alto, California, 94305

Other trials in United States

About this study

Official title: A Phase Ib/II, Single Center, Open-Label, Safety and Efficacy Study to Improve Anemia in Subjects on Enasidenib With Lower Risk Myelodysplastic Syndrome and Non-proliferative Chronic Myelomonocytic Leukemia Without an IDH2 Mutation

This is a phase 1b/2, open-label, single arm study to evaluate if enasidenib is safe and effective in improving anemia and decreasing transfusion needs in subjects diagnosed with lower risk myelodysplastic syndrome (MDS) or nonproliferative chronic myelomonocytic leukemia (CMML) without a mutation in isocitrate dehydrogenase type 2 (IDH2 wildtype). Other objectives include assessment of improvements in platelet production and characterization of the mechanism of action of enasidenib in enhancing endogenous erythropoiesis.

Read the detailed description

Primary Objective(s)- To determine the efficacy (response rate) of enasidenib in improving anemia and decreasing RBC transfusion dependence.

Secondary Objective(s)- To determine the tolerability, safety and durability of the erythroid response and identify laboratory parameters as clinical markers of response.

Being tested
Enasidenib mesylat dose escalation
Medical subject headings
Leukemia, Leukemia, Myeloid, Leukemia, Monocytic, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Enasidenib mesylat dose escalationDrug

    Also called (Idhifa, AG 221)

    Subjects will participate dose escalation with a starting dose of 100 mg. Enasidenib will be self administered orally and daily.

Study groups in NCT05282459
Study groupRoleWhat they receive
Enasidenib mesylatExperimentalParticipants will self administer the enasidenib orally everyday.
Receives: Drug: Enasidenib mesylat dose escalation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Clinical Response: Hematological Improvement - Erythroid (HI-E)

    Clinical response was assessed as the number of participants achieving a hematological improvement - erythroid (HI-E). Participants were characterized and stratified as nontransfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows.

    • NTD = greater than or equal to 2 consecutive Hb measurements, greater than or equal to 1.5 g/dL for a period of minimum 8 week in an observation period of 16 to 24 week compared to the lowest mean of 2 Hb measurements
    • LTB = 0 units of RBC transfusions
    • HTB = greater than or equal to 4 unit or greater than or equal to 50% reduction in RBC transfusions

    Measured: 16 weeks

Secondary outcomes (6)

  1. Related Adverse Events

    Toxicity was assessed as the number of related non-serious adverse events and related serious adverse events (SAEs) reported by dose level (Cohort A or Cohort B) for the 12-cycle treatment period plus follow-up.

    Measured: 12 months

  2. Time to Hematological Improvement - Erythroid (HI-E)

    Time to hematological improvement - erythroid (HI-E) was assessed as the time from first dose of enasidenib to the first observed hemoglobin response. Participants will be characterized and stratified as non-transfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows.

    • NTD = greater than or equal to 2 consecutive Hb measurements, greater than or equal to 1.5 g/dL for a period of minimum 8 week in an observation period of 16 to 24 week compared to the lowest mean of 2 Hb measurements
    • LTB = 0 units of RBC transfusions
    • HTB = greater than or equal to 4 unit or greater than or equal to 50% reduction in RBC transfusions

    Measured: 16 weeks

  3. Duration of Hematological Improvement - Erythroid (HI-E)

    Duration of Hematological Improvement - Erythroid (HI-E) will be assessed as the time from recorded response to loss of response. Participants will be characterized and stratified as nontransfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows.

    NTD = greater than or equal to 2 consecutive Hb measurements, greater than or equal to 1.5 g/dL for a period of minimum 8 week in an observation period of 16 to 24 week compared to the lowest mean of 2 Hb measurements LTB = 0 units of RBC transfusions HTB = greater than or equal to 4 unit or greater than or equal to 50% reduction in RBC transfusions The outcome will be reported as the number of participants that achieve the response, a number without dispersion.

    Measured: 16 weeks

  4. Clinical Response: Hematological Improvement - Platelets (HI-P)

    Clinical response for platelets was assessed as the number of participants achieving a hematological improvement - platelets (HI-P). Participants will be characterized and stratified as platelets < or ≥ 20 x 10^9/L, with response defined as follows.

    • < 20 x 10^9/L = increase in platelets from < 20 x 10^9/L to > 20 x 10^9/L AND by ≥ 100%
    • ≥ 20 x 10^9/L = absolute increase in platelets of 30 x 10^9/L The outcome will be reported as the number of participants that achieve the response, a number without dispersion.

    Measured: 8 weeks

  5. Clinical Response: Hematological Improvement - Neutrophils (HI-N)

    Clinical response for neutrophils was assessed as the number of participants achieving a hematological improvement - neutrophils (HI-N). Response was defined as an absolute increase in neutrophils > 0.5 × 10^9/L that was also an increase of ≥ 100%.

    Measured: 8 weeks

  6. Red Blood Cell (RBC) Transfusion Independence (RBC TI)

    Clinical response for red blood cells was assessed as the number of participants who were transfusion dependent that achieve red blood cell (RBC) transfusion independence (RBC TI) for for 8 weeks or longer.

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Mar 2022
  2. Study start12 Jan 2022
  3. Primary completion3 Dec 2024
  4. Study completion3 Mar 2025

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
17 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05282459
Sponsor's protocol ID
IRB-62692
Other IDs
HEM0056, NCI-2022-02837
Status last verified
Apr 2026
First submitted
7 Mar 2022
Last updated
3 Sep 2026
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Enasidenib mesylat dose escalation in people with Leukemia and Leukemia, Myeloid, run by Tian Yi Zhang 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 26 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: finished, as of the sponsor's last update on 3 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 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Yes. Results are posted in the registry (first posted 17 Apr 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Clinical Response: Hematological Improvement - Erythroid (HI-E) (time frame: 16 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05282459.

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 3 Sep 2026.

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