Tests eltanexor in adults with returning NPM1-mutated acute myeloid leukemia
Tests eltanexor for safety and effect in adults with returning NPM1-mutated acute myeloid leukemia.
Plain-language title generated from the registry record
Registry title: A phase 2A pilot study to assess the safety and efficacy of eltanexor in relapsed/refractory NPM1-mutated acute myeloid leukemia
Key facts
- Where
- Ancona, Catania, and Perugia, ItalySee the 3 sites
- Phase
- Phase 2
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for older adults with acute myeloid leukemia (AML) that has an NPM1 gene change and has come back or did not respond to earlier treatment.
- Age
- 60 years or older.
- Sex
- Men and women. Women must be postmenopausal.
- Disease stage
- AML with an NPM1 mutation that has come back (relapsed) or did not respond (refractory). Life expectancy of at least 3 months. White blood cell count of 10 x 109/L or lower. Cannot have a bone marrow transplant.
- Earlier treatment
- Must have had at least one venetoclax-based regimen, unless it is not allowed. If they could have had intensive chemotherapy at diagnosis, they must also have had at least one line of standard chemotherapy. With a FLT3 mutation, at least one FLT3 inhibitor is also needed. At least 14 days since the last cytotoxic treatment (except hydroxyurea) and 7 days since the last FLT3 inhibitor.
- Biomarker or test result
- NPM1-mutated AML. Liver tests (bilirubin, AST, ALT) and kidney function (creatinine clearance of 30 mL/min or higher) must be adequate.
Main reasons for not taking part
- Major surgery within 4 weeks before the first treatment day
- Serious heart disease, or a heart attack or unstable angina within 3 months
- Severe infection needing antibiotics, antivirals or antifungals by injection or drip within 1 week
- Leukemia known to involve the brain or spinal cord, known HIV, or active hepatitis A, B or C
- Bowel disease or uncontrolled vomiting or diarrhea that could affect how the drug is absorbed
Read by machine on 12 November 2026.
Where in Italy
- Azienda Ospedaliero-Universitaria delle MarcheAncona, ItalyTaking people
- Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di CataniaCatania, ItalyTaking people
- University Hospital Of PerugiaPerugia, ItalyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record
Print a page for my doctorTrial details
- Who runs it
- Azienda Ospedaliero Universitaria Delle Marche
- Trial code
- EU 2024-514724-16-01
- Record updated
- 13 April 2026
- Original description
To evaluate the antileukemic activity of eltanexor in relapsed/refractory NPM1-mutated AML To evaluate the safety and tolerability of eltanexor in relapsed/refractory NPM1-mutated AMLRead full original descriptionShow less
A phase 2A pilot study to assess the safety and efficacy of eltanexor in relapsed/refractory NPM1-mutated acute myeloid leukemia
To evaluate the antileukemic activity of eltanexor in relapsed/refractory NPM1-mutated AML To evaluate the safety and tolerability of eltanexor in relapsed/refractory NPM1-mutated AML
Inclusion: Written informed consent signed prior to any screening procedures and in accordance with local, and institutional guidelines; Ability to swallow oral study medication; Adequate hepatic function: a) total bilirubin ≤2 times the upper limit of normal (ULN) (except patients with Gilbert’s syndrome [hereditary indirect hyperbilirubinemia]. b) aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3 times ULN (except patients with known liver involvement of their tumor who must have their AST and ALT ≤5.0 times ULN); Adequate renal function: estimated creatinine clearance of ≥30 mL/min, calculated using the formula of Cockcroft and Gault (140-Age) × Mass (kg)/(72 × creatinine mg/dL); multiply by 0.85 if female; Female patients must be postmenopausal; Male patients must use an effective barrier method of contraception if sexually active. Effective methods of contraception must be used throughout the study and for 6 months following the last dose; Age ≥60 years; Documented diagnosis of relapsed or refractory NPM1-mutated AML; Life expectancy of at least 3 months at the time of screening; Patient must fall into one of these two categories: a) Patients eligible for standard treatment (intensive chemotherapy) at diagnosis that have received at least one line of standard chemotherapy and one venetoclax-based regimen, unless contraindicated. Additionally, patients with concomitant FLT3 mutations must have received at least one FLT3 inhibitor, unless contraindicated. b) Patients not eligible for standard treatment at diagnosis that have received at least one venetoclax-based regimen, unless contraindicated. Additionally, patients with concomitant FLT3 mutations must have received at least one FLT3 inhibitor, unless contraindicated; Ineligibility for bone marrow transplantation at the time of screening; White blood cell count ≤ 10 x 109/L; A washout time of at least 14 days from the last cytotoxic treatment (except hydroxyurea) and 7 days from the last FLT3 inhibitor; Eastern Cooperative Oncology Group (ECOG) performance status of <2.
Exclusion: Major surgery within 4 weeks before C1D1; Impaired cardiac function or clinically significant cardiac diseases, including any of the following: a) Unstable angina or acute myocardial infarction ≤3 months prior to C1D1 b) Clinically significant heart disease (e.g., symptomatic congestive heart failure [e.g., >NYHA Class 2]; uncontrolled arrhythmia, or hypertension; history of labile hypertension or poor compliance with an antihypertensive regimen); Uncontrolled active severe systemic infection requiring parenteral antibiotics, antivirals, or antifungals within 1 week prior to C1D1; Patients unwilling to comply with the protocol including required biopsies and sample collections required to measure disease; Known history of human immunodeficiency virus (HIV); Known, active hepatitis A, B, or C infection; or known to be positive for HCV RNA or HBsAg (HBV surface antigen); Patients with gastrointestinal tract disease (or uncontrolled vomiting or diarrhea) that could interfere with the absorption of eltanexor; Serious psychiatric or medical conditions that, in the opinion of the Investigator, could interfere with treatment, compliance, or the ability to give consent; Known CNS involvement by AML; Female patients with childbearing potential.