RECRUITING

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

Conditions

Clonal Cytopenia of Undetermined Significance CCUS Clonal Cytopenia of Undetermined Significance CCUS IDH2 Enasidenib

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2024-10-10

Study Completion:2026-10-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06240754

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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).	* 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.

Inclusion Criteria

Exclusion 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).

* 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.

Contacts and Locations

Study Contact

Kelly Bolton, M.D., Ph.D.

CONTACT

314-273-5711

bolton@wustl.edu

Principal Investigator

Kelly Bolton, M.D., Ph.D.

PRINCIPAL_INVESTIGATOR

Washington University School of Medicine

Study Locations (Sites)

Washington University School of Medicine

Saint Louis, Missouri, 63110

United States

Collaborators and Investigators

Sponsor: Washington University School of Medicine

Kelly Bolton, M.D., Ph.D., PRINCIPAL_INVESTIGATOR, Washington University School of Medicine

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-10-10

Study Completion Date2026-10-31

Study Record Updates

Study Start Date2024-10-10

Study Completion Date2026-10-31

Terms related to this study

Keywords Provided by Researchers

Clonal Cytopenia of Undetermined Significance
CCUS
IDH2
Enasidenib

Additional Relevant MeSH Terms

Clonal Cytopenia of Undetermined Significance
CCUS Clonal Cytopenia of Undetermined Significance