Study on the Effectiveness and Safety of Imetelstat for Patients with High-Risk Myelodysplastic Syndromes or Acute Myeloid Leukemia After HMA Therapy Failure

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What is this study about?

This clinical trial is focused on studying the effectiveness and safety of a treatment called Imetelstat for patients with certain blood disorders. The diseases being studied are Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS). These are conditions where the bone marrow does not produce enough healthy blood cells. The trial is specifically for patients whose conditions have not improved with a type of treatment known as hypomethylating agents (HMA). The main goal of the study is to see how well Imetelstat works in treating these diseases when previous treatments have not been successful.

Participants in the study will receive Imetelstat, which is given as a solution through an intravenous infusion, meaning it is delivered directly into the bloodstream. The study will also involve other medications, such as Diphenhydramine and Hydrocortisone, which are used to manage symptoms and side effects. Diphenhydramine is an antihistamine that can help with allergic reactions, while Hydrocortisone is a corticosteroid that can reduce inflammation. The study will last for a period of up to nine months, during which the participants will be closely monitored to assess the treatment’s effects and any side effects.

Throughout the study, participants will have regular check-ups to monitor their health and the progress of their treatment. The study aims to gather information on how well the treatment works and how it affects the overall health and quality of life of the participants. This information will help in understanding the potential benefits and risks of using Imetelstat for treating AML and MDS in patients who have not responded to other treatments.

1 joining the study

Upon joining the study, you will be required to provide a signed written informed consent. This document confirms your understanding and agreement to participate in the clinical trial.

You will undergo a series of initial tests to ensure eligibility, including blood tests and a pregnancy test if applicable. These tests will confirm that your health status meets the study’s requirements.

2 treatment initiation

The treatment involves the administration of imetelstat sodium through an intravenous infusion. This means the medication will be delivered directly into your bloodstream through a vein.

You will also receive diphenhydramine hydrochloride and hydrocortisone intravenously to help manage any potential side effects. The specific dosage and frequency will be determined by the study team based on your individual needs.

3 treatment cycles

The treatment is organized into cycles. Each cycle lasts for a specific period, during which you will receive the medication as scheduled by the study team.

You will be monitored closely throughout each cycle to assess your response to the treatment and to manage any side effects that may occur.

4 regular assessments

Throughout the study, you will undergo regular assessments to evaluate the effectiveness of the treatment. These assessments may include blood tests, physical examinations, and questionnaires about your health and quality of life.

The study team will use these assessments to determine your overall response to the treatment and to make any necessary adjustments to your care plan.

5 end of treatment

At the end of the treatment period, you will have a final set of assessments to evaluate your health status and the outcomes of the study.

You will be provided with information about any follow-up care or additional monitoring that may be required after the study concludes.

Who Can Join the Study?

Must provide a signed written informed consent, which means you agree to participate in the study after understanding all the details.
Must have an ECOG performance status of 0-2, which is a scale used to assess how your disease affects your daily living abilities. A score of 0 means fully active, and 2 means you are up and about more than half of waking hours.
Must have certain blood test results within specific limits:
- AST, ALT, and ALP (liver enzymes) should be no more than 2.5 times the normal level.
- Serum creatinine (a kidney function marker) should be no more than 2 times the normal level.
- Total bilirubin should be no more than 3 times the normal level, and direct bilirubin no more than 2 times the normal level, unless due to specific conditions like Gilbert’s syndrome.
Must have records of blood counts and transfusion events for the past 16 weeks.
Women who can have children must use a highly effective method of birth control during the study and for 3 months after the last dose. This includes options like hormonal contraception, an intrauterine device (IUD), or having a partner who has had a vasectomy.
Women who can have children must have a negative pregnancy test before starting the study and agree to regular testing during the study.
Men who are sexually active with women who can have children must use a barrier method of birth control, like a condom, and must not donate sperm during the study and for 3 months after the last dose.
Must be 18 years or older at the time of the first screening.
Must be able to follow the study visit schedule and other study requirements.
Must have an initial diagnosis of AML or MDS according to the WHO 2016 classification.
Must have at least one cytopenia, which means low levels of certain blood cells, such as:
- ANC (absolute neutrophil count) less than 1800/µL
- Platelet count less than 100,000/µL
- Hemoglobin less than 10 g/dL
Must have not responded to or relapsed after specific treatments for AML or MDS in the past two years, or be intolerant to these treatments.
Must not be eligible for a stem cell transplant from another person.
Must have 5% or more bone marrow blasts at screening, which are immature blood cells found in the bone marrow.
Must have stopped all other treatments for AML/MDS at least 14 days before starting the study, except for certain supportive treatments like G-CSF and erythropoietin.

Who Cannot Join the Study?

Patients who have not failed or are not resistant to treatment with a type of medication called hypomethylating agents (HMA). These are drugs used to treat certain blood disorders.
Patients who are not diagnosed with AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes).
Patients who are not within the specified age range for the study.
Patients who belong to a vulnerable population, which means they might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country
University Hospital Jena KöR	Jena	Germany
Universitaet Leipzig	Leipzig	Germany
Oncopole Claudius Regaud	Toulouse	France

Other Sites

Site Name	City	Country
Marien Hospital Duesseldorf GmbH	Duesseldorf	Germany
Centre Hospitalier Universitaire De Nice	Nice	France
Centre Hospitalier Universitaire De Nantes	Nantes	France
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)	Munich	Germany
Awmfkvodyc Pdqlkjby Hyaznuob Da Pyjyr	Paris	France

Trial status

Country	Status	Recruitment Start
France	Not yet recruiting	31.12.2022
Germany	Not yet recruiting	31.12.2022

Trial locations

Investigated drugs:

Imetelstat is a medication being studied for its potential to help patients with certain blood disorders, specifically myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). These conditions often involve the bone marrow not producing enough healthy blood cells. Imetelstat works by targeting and blocking a specific enzyme that cancer cells need to grow. This could help slow down or stop the progression of the disease, especially in patients who have not responded well to other treatments. The goal of using Imetelstat in this trial is to see if it can improve the health and quality of life for patients who have not had success with their previous therapies.

Investigated diseases:

Acute Myeloid Leukemia – Acute Myeloid Leukemia (AML) is a type of cancer that starts in the blood-forming cells of the bone marrow and quickly progresses to affect the blood. It is characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with the production of normal blood cells. As the disease progresses, it can spread to other parts of the body, including the lymph nodes, liver, spleen, and central nervous system. Symptoms may include fatigue, fever, frequent infections, and easy bruising or bleeding. The progression of AML can vary, but it typically advances quickly without treatment.

Myelodysplastic Syndromes – Myelodysplastic Syndromes (MDS) are a group of disorders caused by poorly formed or dysfunctional blood cells. These syndromes occur when the blood-forming cells in the bone marrow are damaged, leading to a decrease in the number of healthy blood cells. Over time, MDS can progress to more severe forms of blood disorders, including acute myeloid leukemia. Symptoms often include fatigue, shortness of breath, and increased risk of bleeding and infections due to low blood cell counts. The progression of MDS can vary widely among individuals, with some experiencing a slow progression and others a more rapid decline.

Trial ID:

2022-500721-32-00

Protocol code:

IMpress_001

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Effectiveness and Safety of Imetelstat for Patients with High-Risk Myelodysplastic Syndromes or Acute Myeloid Leukemia After HMA Therapy Failure

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?