Old drug, new hope: enasidenib may ease anemia in blood cancer patients
NCT ID NCT05282459
First seen Jun 27, 2026 · Last updated Sep 04, 2026 · Updated 2 times
Summary
This early-phase trial tested the drug enasidenib in 17 people with low-risk myelodysplastic syndrome or a related leukemia who had anemia and did not have a specific gene mutation. The goal was to see if the drug could safely improve red blood cell counts and reduce the need for transfusions. Researchers also looked at how the drug works to boost red blood cell production.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Enasidenib (Idhifa, AG 221)
- What this could lead to
- If successful, enasidenib could offer a new way to improve anemia and reduce blood transfusions for people with certain blood cancers.
- What could go wrong
- This is a very early, small study (17 people) with no placebo group, so results may not be reliable or apply to everyone. Side effects are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
17 people
The number who actually took part.
- Started
-
Jan 2022
- Finished
-
Mar 2025
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. 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) 2. No disease-modifying therapy (HMA, hydrea) within 2 months of starting study 3. Age ≥ 18 years of age 4. ECOG ≤ 3 5. Negative for IDH2 mutation by NGS or multiplex PCR (SNaPshot) 6. 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. 7. Stated willingness to comply with all study procedures and availability for the duration of the study 8. Ability to take oral medication and be willing to adhere to the medication regimen. 9. 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 10. For males of reproductive potential: agreement to use of condoms 11. 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 12. Ability to understand and the willingness to sign the IRB approved informed consent document. 13. Women of childbearing potential must have negative urine or serum pregnancy test Exclusion Criteria: 1. Use of concurrent other erythropoietic agents (including epoetin, darbepoetin), G-CSF within 30 days of study enrollment 2. Less than 3 months of life expectancy 3. Significant cardiac disease (NYHA Class IV congestive heart failure, or unstable angina or myocardial infarction within the last 6 months 4. Harbor IDH2 somatic mutations by NGS or PCR 5. Pregnant or breast feeding 6. Any uncontrolled bacterial, fungal, viral or other infection. 7. 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. 8. 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. 9. 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 10. Pregnant or breast feeding
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Stanford Cancer Institute
Palo Alto, California, 94305, United States
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