Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2A Decentralized Trial

Clonal Cytopenia of Undetermined Significance Clinical Trial

Official title:

A Pilot Study of Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2: A Decentralized Trial

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06240754
• Other study ID #: 24-x022
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: June 30, 2024
• Est. completion date: June 30, 2026

Study information

• Verified date: May 2024
• Source: Washington University School of Medicine
• Contact: Kelly Bolton, M.D., Ph.D.
• Phone: 314-273-5711
• Email: bolton[@]wustl.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 15
• Est. completion date: June 30, 2026
• Est. primary completion date: May 31, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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.

• 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 4 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 (or that of legally authorized representative, if applicable).

Exclusion Criteria:

• 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.

• 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.

Related Conditions & MeSH terms

• Anemia
• Clonal Cytopenia of Undetermined Significance
• Cytopenia
• Leukopenia
• Thrombocytopenia

Intervention

Drug:
• Enasidenib
Provided by BMS.

Locations

Country	Name	City	State
United States	Washington University School of Medicine	Saint Louis	Missouri

Sponsors (3)

Lead Sponsor	Collaborator
Washington University School of Medicine	Bristol-Myers Squibb, Damon Runyon Cancer Research Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Up to 18 cycles (each cycle is 28 days) of treatment (up to approximately 17 months)
Secondary	Toxicity as measured by the number of adverse events experienced by participant	Measured by CTCAE v 5.0	From start of treatment through 30 days after the last day of treatment (up to approximately 18 months)
Secondary	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.	Baseline, day 1 of cycles 3/6/9/12/15 (each cycle is 28 days), and end of treatment (up to approximately 17 months)
Secondary	Acute myeloid leukemia (AML-free) or myelodysplastic syndrome (MDS)-free survival		From start of treatment through completion of follow-up (up to approximately 18 months)

External Links

•   Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine