A Study of Enasidenib in People With Clonal Cytopenia of Undetermined Significance

Phase 1

Active, not recruiting

• Conditions: Clonal Cytopenia of Undetermined Significance; CCUS Clonal Cytopenia of Undetermined Significance

• Registration Number: NCT05102370
• Lead Sponsor: Memorial Sloan Kettering Cancer Center

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-10-20
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: Active, not recruiting
• Sex: All
• Target Recruitment: 4

Inclusion Criteria

Inclusion Criteria:<br><br> - Age =18 years at the time of signing the informed consent form.<br><br> - Willing and able to adhere to the study visit schedule and other protocol<br> requirements.<br><br> - Unexplained cytopenia for at least 6 months. Cytopenia is defined as the presence of<br> =1 blood count indexes below the following thresholds:<br><br> - Hgb <10 g/dL<br><br> - ANC <1.8 × 10^9/L<br><br> - Platelets <100 × 10^9/L<br><br> - Results of bone marrow biopsy within 1 month of study entry (screening bone marrow<br> biopsy) must not indicate hematologic disease.<br><br> - IDH2 gene mutation (R140 or R172), performed locally, at a frequency >2%.<br><br> - ECOG performance status of 0-2.<br><br> - Adequate organ function, defined as:<br><br> - Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase<br> (AST/SGOT) and alanine aminotransferase (ALT/SGPT) =3x upper limit of normal<br> (ULN), unless the subject has Gilbert's syndrome.<br><br> - Serum total bilirubin <1.5x ULN. Higher levels are acceptable if these can be<br> attributed to ineffective erythropoiesis. In these cases, approval from the<br> study Principal Investigator is required.<br><br> - Creatinine clearance greater than 50 mL/min based on the Cockroft-Gault<br> glomerular filtration rate estimation.<br><br> - Patients being enrolled on study on the basis of anemia, will only be eligible if<br> folate, B12, serum iron, serum ferritin, total iron binding capacity, haptoglobin<br> and peripheral smear within normal limits<br><br> - Women of childbearing potential may participate provided they have a negative serum<br> pregnancy test at screening and a negative serum or urine pregnancy test within 72 h<br> of starting treatment.<br><br> - Women of childbearing potential (WOCBP) and males with partners who are WOCBP must<br> agree to abstain from sexual intercourse or to use 1 highly effective form of<br> contraception during the study and for at least 4 months following the last dose of<br> enasidenib. Males with partners who are WOCBP must agree to use a barrier method.<br><br>Exclusion Criteria:<br><br> - Active malignancy defined as >1-cm disease on most recent CT scan in the past 6<br> months or recent history of cancer (i.e. within the past 5 years) with >50% chance<br> of cancer recurrence in the next 5 years.<br><br> - Current or prior history of hematologic malignancy.<br><br> - Therapy (including maintenance therapy) for solid-tumor malignancy within the last 6<br> months.<br><br> - Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit<br> the ingestion or gastrointestinal absorption of drugs administered orally.<br><br> - Active uncontrolled systemic fungal, bacterial, or viral infection (defined as<br> ongoing signs/symptoms related to the infection without improvement despite<br> appropriate antibiotics, antiviral therapy, and/or other treatment).<br><br> - Positive direct Coombs test<br><br> - Evidence of hypersplenism on physical exam<br><br> - Pregnant or lactating (women).

Exclusion Criteria

Not provided

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Best Response