Study on the Safety and Effectiveness of Eltanexor for Patients with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

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What is this study about?

This clinical trial is focused on studying a type of blood cancer called Acute Myeloid Leukemia (AML), specifically in patients whose cancer has returned or has not responded to previous treatments. The study is testing a new medication called Eltanexor, also known by its code name KPT-8602. Eltanexor is taken in the form of a tablet and is being investigated for its ability to fight against this type of leukemia and to understand how safe and tolerable it is for patients.

The purpose of the study is to evaluate how well Eltanexor works in treating patients with a specific mutation in their leukemia called NPM1-mutated AML. Participants in the study will take Eltanexor orally, meaning they will swallow the tablets. The study will monitor the effects of the medication over a period of time to see how the leukemia responds and to check for any side effects. The study aims to gather information on the response rate of the leukemia to the treatment and to observe any significant side effects that may occur.

Throughout the study, participants will be closely monitored by healthcare professionals to ensure their safety and to collect data on the effectiveness of Eltanexor. The study is designed to provide valuable insights into the potential benefits and risks of using Eltanexor for treating relapsed or refractory NPM1-mutated AML, contributing to the development of new treatment options for this challenging condition.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing medical history and current health status.

Eligibility criteria include the ability to swallow oral medication, adequate liver and kidney function, and a documented diagnosis of relapsed or refractory NPM1-mutated acute myeloid leukemia.

2 medication administration

The study involves taking eltanexor, which is administered in tablet form for oral use.

The dosage and frequency of eltanexor are determined by the study protocol and will be explained during the trial.

3 monitoring and evaluation

Regular monitoring is conducted to assess the safety and effectiveness of the treatment. This includes blood tests and other evaluations as required.

The primary goal is to evaluate the antileukemic activity of eltanexor and monitor any side effects.

4 response assessment

The response to the treatment is assessed by the end of cycle 2. This includes measuring the response rate and any adverse events.

The study aims to determine the overall response rate and the occurrence of any significant side effects.

5 completion of study

The study is estimated to conclude by June 2026. Participants will be informed about the outcomes and any further steps if necessary.

Continued monitoring may be required after the study to ensure long-term safety and effectiveness.

Who Can Join the Study?

Sign a written informed consent form before any screening procedures, following local and institutional guidelines.
Be able to swallow the study medication in pill form.
Have adequate liver function, which means:
- Total bilirubin levels should be no more than 2 times the normal limit, except for patients with a condition called Gilbert’s syndrome.
- Levels of liver enzymes called AST and ALT should be no more than 3 times the normal limit, unless the liver is affected by the tumor, in which case they should be no more than 5 times the normal limit.
Have adequate kidney function, with an estimated creatinine clearance of at least 30 mL/min. This is calculated using a formula that considers age, weight, and creatinine levels in the blood.
Female patients must be postmenopausal, meaning they have stopped having menstrual periods.
Male patients must use an effective barrier method of contraception if they are sexually active. This should be used throughout the study and for 6 months after the last dose.
Be at least 60 years old.
Have a documented diagnosis of relapsed or refractory NPM1-mutated AML, which is a type of blood cancer.
Have a life expectancy of at least 3 months at the time of screening.
Meet one of the following treatment history criteria:
- For patients eligible for standard treatment at diagnosis: must have received at least one line of standard chemotherapy and one venetoclax-based regimen, unless not suitable. If they have FLT3 mutations, they must have received at least one FLT3 inhibitor, unless not suitable.
- For patients not eligible for standard treatment at diagnosis: must have received at least one venetoclax-based regimen, unless not suitable. If they have FLT3 mutations, they must have received at least one FLT3 inhibitor, unless not suitable.
Not be eligible for bone marrow transplantation at the time of screening.
Have a white blood cell count of no more than 10 x 10⁹/L.
Have a washout period of at least 14 days from the last cytotoxic treatment (except hydroxyurea) and 7 days from the last FLT3 inhibitor.
Have an Eastern Cooperative Oncology Group (ECOG) performance status of less than 2, which is a measure of the patient’s ability to perform daily activities.

Who Cannot Join the Study?

Patients who do not have Acute Myeloid Leukemia cannot participate. Acute Myeloid Leukemia is a type of cancer that affects the blood and bone marrow.
Patients who do not have the specific genetic mutation called NPM1 mutation in their leukemia cannot participate. This is a change in the DNA that can affect how the disease behaves.
Patients who have not experienced a return of their leukemia after treatment, known as relapsed, or whose leukemia has not responded to treatment, known as refractory, cannot participate.
Patients who are not within the specified age range for the study cannot participate. The study is open to adults.
Patients who are part of a vulnerable population, such as those unable to give consent, cannot participate.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania	Catania	Italy
Azienda Ospedaliera Di Perugia	Perugia	Italy
Universita’ Politecnica Delle Marche	Ancona	Italy

Trial status

Country	Status	Recruitment Start
Italy	Recruiting	01.07.2023

Trial locations

Investigated drugs:

ELTANEXOR

Eltanexor is a medication being studied for its potential to treat acute myeloid leukemia (AML) that has returned or is not responding to treatment. This medication is specifically being tested in patients whose leukemia has a mutation in the NPM1 gene. The study aims to determine how effective Eltanexor is in reducing leukemia cells and to assess its safety and how well patients can tolerate it.

Acute Myeloid Leukemia – Acute Myeloid Leukemia (AML) is a type of cancer that starts in the blood-forming cells of the bone marrow. It leads to the rapid growth of abnormal white blood cells, which accumulate in the bone marrow and interfere with the production of normal blood cells. This results in symptoms such as fatigue, frequent infections, and easy bruising or bleeding. AML progresses quickly and requires prompt medical attention. The disease can affect people of all ages but is more common in adults. It is characterized by various genetic mutations, including the NPM1 mutation, which can influence the disease’s behavior and response to treatment.

Trial ID:

2024-514724-16-01

Protocol code:

ELENA-01

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Safety and Effectiveness of Eltanexor for Patients with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?