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

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

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 conditions affect the blood and bone marrow, leading to issues like anemia and increased risk of infections. The trial is specifically for patients whose previous treatments with hypomethylating agents (HMA) have not been successful.

The treatment being tested, Imetelstat, is a type of medication known as a telomerase inhibitor, which is given as a solution through an intravenous infusion. This means it is administered directly into the bloodstream. The study will also involve other medications like Hydrocortisone, a corticosteroid, and Diphenhydramine, an antihistamine, which may be used to manage side effects or other symptoms during the trial. Some participants may receive a placebo instead of the active treatment to help compare the effects.

The purpose of the study is to determine how well Imetelstat works in treating patients with AML and MDS who have not responded to previous treatments. Participants will receive the treatment over a period of several months, with regular check-ups to monitor their health and the effects of the medication. The study aims to provide valuable information on the potential benefits and risks of using Imetelstat for these conditions.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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 must be able to adhere to the study visit schedule and other protocol requirements. This means attending all scheduled appointments and following the study guidelines.

  2. Step 2

    Screening and initial assessments

    You will undergo initial assessments to confirm your eligibility. This includes verifying your diagnosis of acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) and checking for specific blood cell counts.

    A series of laboratory tests will be conducted to ensure your biochemical levels are within the required limits. This includes tests for liver enzymes, kidney function, and bilirubin levels.

  3. Step 3

    Treatment initiation

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

    You will receive the infusion according to the study's schedule, which will be explained to you by the medical team.

  4. Step 4

    Ongoing treatment and monitoring

    Throughout the study, you will continue to receive imetelstat infusions as per the protocol. The frequency and duration of these infusions will be determined by the study guidelines.

    Regular monitoring will be conducted to assess your response to the treatment. This includes blood tests and other evaluations to track your health and any changes in your condition.

  5. Step 5

    Safety and efficacy assessments

    The study aims to evaluate the effectiveness and safety of imetelstat in treating your condition. This involves assessing the overall response rate after four months of treatment.

    Your overall survival and progression-free survival will be monitored. This means tracking the time from the start of treatment until any progression of the disease or other significant events.

  6. Step 6

    Completion of the study

    At the end of the study, final assessments will be conducted to evaluate your health status and the outcomes of the treatment.

    You will be informed about the results and any further steps or recommendations based on your participation in the trial.

Who can join the trial?

22 criteria

  • Must provide a signed written informed consent.
  • Must be able to follow the study visit schedule and other study requirements.
  • Must have an initial diagnosis of AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes) according to the WHO 2016 classification.
  • Must have at least one cytopenia, which means low blood cell counts:
    • ANC (Absolute Neutrophil Count) less than 1800/μL
    • Platelet count less than 100,000/μL
    • Hemoglobin less than 10 g/dL
    • Must have experienced failure or intolerance to certain treatments in the past two years, such as:
      • Not achieving a complete or partial response after specific treatment cycles with azacitidine or decitabine, alone or with venetoclax.
      • Relapse after initial response to these treatments.
      • Intolerance to HMA-based therapy (Hypomethylating Agent-based therapy).
      • Not eligible for allogeneic stem cell transplantation, which is a type of bone marrow transplant.
      • Must have 5% or more bone marrow blasts at screening. Blasts are immature blood cells.
      • Must have stopped all other treatments for AML/MDS for at least 14 days, except for G-CSF (a growth factor) and erythropoietin (a hormone that stimulates red blood cell production), which are allowed.
      • Must be relapsed or refractory to, or not eligible for, therapy with approved and available FLT3 or IDH1/IDH2 inhibitors or other approved targeted therapies.
      • Must have an ECOG performance status of 0-2, which is a scale to assess how a disease affects daily living abilities.
      • Must have certain biochemical laboratory test values within specified limits:
        • AST, ALT, and ALP (liver enzymes) no more than 2.5 times the upper limit of normal.
        • Serum creatinine no more than 2 times the upper limit of normal.
        • Total bilirubin no more than 3 times the upper limit of normal and direct bilirubin no more than 2 times the upper limit of normal, unless due to specific conditions like Gilbert’s syndrome.
        • Must have available records of blood counts and transfusion events for the previous 16 weeks.
        • Women of childbearing potential must use a highly effective method of birth control during the study and for 3 months after the end of dosing.
        • Women of childbearing potential must have a negative pregnancy test at screening and agree to regular testing during the study.
        • Men who are sexually active with women of childbearing potential must use a barrier method of birth control and must not donate sperm during the study and for 3 months after the end of dosing.
        • Must be 18 years or older at the first screening.

Who cannot join the trial?

4 criteria

  • Patients who have not failed or are not resistant to treatment with a type of medication called a hypomethylating agent (HMA).
  • 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 group.
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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. By doing this, it may help to slow down or stop the progression of the disease in patients who have not responded well to other treatments based on hypomethylating agents (HMA).

What is already known about the treatment

Imetelstat – Imetelstat is administered intravenously, meaning it is given directly into a vein through an injection. It is currently being studied in clinical trials for its effectiveness and safety in treating patients with high-risk myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who have not responded to or have become resistant to treatments based on hypomethylating agents (HMAs). The main therapeutic use of Imetelstat is to help these patients by targeting and inhibiting a specific enzyme called telomerase, which is often active in cancer cells and helps them to keep dividing. By blocking this enzyme, Imetelstat aims to stop the growth of cancer cells. It is classified as a telomerase inhibitor in pharmacology.

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 often include fatigue, fever, frequent infections, and easy bruising or bleeding. The progression of AML can lead to a decrease in red blood cells, white blood cells, and platelets, causing anemia, increased risk of infection, and bleeding issues. The disease can advance rapidly, requiring prompt medical attention.

  • 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. As MDS progresses, it can result in anemia, frequent infections, and easy bleeding due to low levels of red blood cells, white blood cells, and platelets. The condition can remain stable for years or progress to more severe forms, such as acute myeloid leukemia. Symptoms may include fatigue, shortness of breath, and unusual bruising or bleeding. The progression of MDS varies among individuals, with some experiencing a slow decline in blood cell counts and others facing a more rapid progression.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2022-500721-32-01Protocol codeIMpress_001Estimated enrolment46 patientsSponsorGcp-Service International West GmbH

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