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Study of AG-946 for Treating Anemia in Patients with Lower-Risk Myelodysplastic Syndromes

Fast replyInvestigationalNo 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 a condition called anemia, which is a situation where the body does not have enough healthy red blood cells to carry oxygen. Specifically, the trial is looking at anemia caused by a group of blood disorders known as lower-risk myelodysplastic syndromes (LR-MDS). These syndromes affect the bone marrow and can lead to a shortage of blood cells. The treatment being tested in this study is a medication called AG-946, which is taken as a coated tablet by mouth.

The purpose of the study is to explore how effective and safe AG-946 is for people with anemia due to LR-MDS. The study is divided into two phases. In the first phase, researchers aim to see if the treatment shows promise in helping patients. In the second phase, they will look at whether the treatment can help patients become independent of blood transfusions, meaning they won't need transfusions for a certain period. Participants will take the medication for a maximum of 180 days, and their health will be monitored throughout the study.

Some participants will receive the actual medication, while others may receive a placebo. The study will help determine if AG-946 can improve the condition of those with LR-MDS by increasing their hemoglobin levels, which is a protein in red blood cells that carries oxygen. The trial will also assess any side effects or changes in the participants' health during the study period. This research is important for finding new ways to manage anemia in people with LR-MDS.

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, participants will be informed about the trial's purpose and procedures. Written informed consent is required before any study-related activities begin.

  2. Step 2

    Initial assessment

    Participants will undergo an initial assessment to confirm eligibility. This includes a review of medical history, a physical examination, and laboratory tests to ensure the participant meets the study criteria.

  3. Step 3

    Medication administration

    Participants will receive the study medication, AG-946, in the form of a coated tablet. The medication is taken orally. The dosage and frequency will be determined by the study protocol and communicated to the participant.

  4. Step 4

    Regular monitoring

    Participants will attend regular visits to monitor their health and response to the medication. This includes blood tests to measure hemoglobin levels and other relevant parameters.

  5. Step 5

    Evaluation of outcomes

    The study aims to evaluate the effect of AG-946 on anemia due to lower-risk myelodysplastic syndromes. Participants' progress will be assessed based on changes in hemoglobin levels and transfusion independence.

  6. Step 6

    Completion of study

    Upon completion of the study period, participants will undergo a final assessment. This includes a review of their overall health and any changes observed during the trial.

Who can join the trial?

10 criteria

  • At least 18 years old.
  • Have a documented diagnosis of Myelodysplastic Syndromes (MDS), which is a group of disorders caused by poorly formed or dysfunctional blood cells.
  • Meet the criteria for lower-risk MDS, which means having a risk score of 3.5 or less and less than 5% blasts (immature blood cells) in the bone marrow.
  • For Phase 2a: Either not needing blood transfusions or having a low transfusion burden, which means receiving less than 3 units of red blood cells in the 16 weeks before starting the study drug and no transfusions in the 8 weeks before starting the study drug.
  • For Phase 2b: Having a low or high transfusion burden, which means receiving 3 to 7 units of red blood cells in the 16 weeks before starting the study drug or 8 or more units in the same period.
  • Have a hemoglobin (Hb) concentration of less than 11.0 g/dL for Phase 2a or less than 10.0 g/dL for Phase 2b during the 4-week screening period. Hemoglobin is a protein in red blood cells that carries oxygen.
  • Have an Eastern Cooperative Oncology Group (ECOG) Performance Status score of 0, 1, or 2, which measures the ability to perform daily activities.
  • If taking iron chelation therapy, the dose must have been stable and started at least 56 days before the first dose of the study drug. Iron chelation therapy is used to remove excess iron from the body.
  • Women of childbearing potential must agree to use a highly effective method of contraception or be abstinent from sexual activities that may result in pregnancy during the study and for 28 days after the last dose of the study drug. Men with partners who are women of childbearing potential must agree to use a condom or be abstinent during the study and for 28 days after the last dose of the study drug.
  • Provide written informed consent before any study-related procedures and be willing to comply with all study procedures for the duration of the study.

Who cannot join the trial?

12 criteria

  • Patients with any other type of anemia not related to lower-risk myelodysplastic syndromes cannot participate.
  • Patients who have received any other investigational drug within the last 30 days are excluded.
  • Patients with a history of severe allergic reactions to any medication are not eligible.
  • Patients with uncontrolled high blood pressure (hypertension) cannot join the study.
  • Patients with active infections that require treatment with antibiotics are excluded.
  • Patients with significant heart disease, such as heart failure or recent heart attack, are not eligible.
  • Patients with liver disease that affects liver function are excluded.
  • Patients with kidney disease that affects kidney function cannot participate.
  • Patients who are pregnant or breastfeeding are not eligible for the study.
  • Patients with a history of cancer, except for certain skin cancers, are excluded.
  • Patients who have had a major surgery within the last 4 weeks cannot join the study.
  • Patients with any other medical condition that the study doctors believe would make participation unsafe are excluded.
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Investigated drugs

AG-946 is a medication being studied for its potential to help people with a condition called lower-risk myelodysplastic syndromes, which is a type of blood disorder. This medication is being tested to see if it can improve the symptoms of anemia, a condition where the body doesn't have enough healthy red blood cells. The goal of the study is to find out if AG-946 can help patients become less dependent on blood transfusions, which are often needed to manage anemia. By participating in this trial, researchers hope to learn more about how effective and safe AG-946 is for treating this specific type of anemia.

What is already known about the treatment

AG-946 – AG-946 is administered orally and is currently being studied in clinical trials for its potential use in treating anemia caused by lower-risk myelodysplastic syndromes (LR-MDS). This medication is in the experimental phase, and researchers are evaluating its effectiveness and safety. The main goal is to see if AG-946 can help patients become less dependent on blood transfusions. At the molecular level, AG-946 works by targeting specific pathways in the body to improve red blood cell production. It is classified as a therapeutic agent for blood disorders.

Investigated diseases

Anemia Due to Lower-Risk Myelodysplastic Syndromes – This condition is a type of anemia that occurs when the bone marrow does not produce enough healthy blood cells, specifically red blood cells. It is associated with myelodysplastic syndromes, which are a group of disorders caused by poorly formed or dysfunctional blood cells. In lower-risk cases, the disease progresses slowly, and patients may experience symptoms like fatigue, weakness, and shortness of breath due to the reduced number of red blood cells. Over time, the need for blood transfusions may increase as the body's ability to produce red blood cells continues to decline. The condition is characterized by a low transfusion burden initially, but this can change as the disease progresses. The focus is often on managing symptoms and maintaining quality of life.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2022-500609-42-00Protocol codeAG946-C-002Estimated enrolment131 patientsSponsorAgios Pharmaceuticals Inc.

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