All studies
Active not recruitingPHASE2INTERVENTIONAL

A Study of Tebapivat (AG-946) in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS)

This research is looking into a new medication called tebapivat for individuals living with lower-risk myelodysplastic syndromes (LR-MDS). LR-MDS is a condition affecting the bone marrow, where it doesn't make enough healthy blood cells. People with LR-MDS often need regular blood transfusions to help with their anaemia (low red blood cells). The main goal of this study is to find out if tebapivat can help reduce or even stop the need for these blood transfusions. The study is split into two parts: first, to see if the medicine works as expected, and then to properly measure its effect on reducing transfusions. Participants must be at least 18 years old and meet specific health criteria related to their MDS and transfusion history.

At a glance

Status
Active not recruiting
Phase
PHASE2
Sponsor
Agios Pharmaceuticals, Inc.
Enrolment target
87
Start
07 Nov 2022
Estimated completion
01 Mar 2029

What is this study about?

This study is about a new medicine called tebapivat. It's being tested for people who have a condition called lower-risk myelodysplastic syndromes, or LR-MDS. MDS is a type of bone marrow disorder where your body doesn't produce enough healthy blood cells. This can lead to anaemia, which means you have low red blood cells and might feel tired or breathless. Many people with LR-MDS need regular blood transfusions to help manage their anaemia.

The main purpose of this research is to see if tebapivat can help your body make more red blood cells naturally. If it does, it could mean you wouldn't need as many blood transfusions, or perhaps even stop needing them altogether. This could significantly improve your quality of life.

The study is happening in two stages. The first stage is to get an early idea if the medicine works in people with LR-MDS. The second stage will then look more closely at how well tebapivat helps people become 'transfusion independent', which means they no longer need blood transfusions.

Key takeaways

  • This study is testing a new drug, tebapivat, for lower-risk MDS.
  • The main goal is to reduce or eliminate the need for blood transfusions.
  • Participants must be aged 18 or older with a confirmed LR-MDS diagnosis.
  • Eligibility depends on your recent blood transfusion history and haemoglobin levels.
  • It's a Phase 2 study, meaning it's still in the earlier stages of testing.
  • Participation involves taking the study drug and regular health checks.

Who may be eligible?

To join this study, people need to be at least 18 years old and have a confirmed diagnosis of lower-risk myelodysplastic syndromes (LR-MDS). Your doctor will have classified your condition as 'lower-risk' and checked that you have less than 5% abnormal cells in your bone marrow.

Another important part of joining is your recent history with blood transfusions. You could be eligible if you haven't had many transfusions recently, or none at all. Specifically, if you've had fewer than 3 blood transfusions in the last 16 weeks and none in the last 8 weeks, or if you've had between 3 and 7 transfusions in the last 16 weeks but fewer than 4 in the last 8 weeks, you might be able to take part. Your blood count (haemoglobin) also needs to be below a certain level.

Your general health and ability to do daily activities, as assessed by your doctor, are also taken into account. If you are taking medication to remove excess iron from your body, it needs to have been at a steady dose for at least 8 weeks. For safety, women who could become pregnant and men with partners who could become pregnant will need to use effective contraception throughout the study.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

  1. Are you 18 years old or older?
  2. Have you been diagnosed with lower-risk myelodysplastic syndromes (LR-MDS)?
  3. Have you had fewer than 8 blood transfusions in the last 16 weeks, and perhaps none in the last 8 weeks?
  4. Is your haemoglobin (red blood cell count) currently on the low side?
  5. Are you generally well enough to take part in a study (as judged by your doctor)?
  6. If relevant, are you willing to use contraception during the study?
Answer every question to see your result.

What does participation involve?

The detailed involvement in this study isn't fully described here, but generally, taking part in a clinical trial like this would involve regular visits to the clinic. During these visits, you would have medical check-ups, blood tests, and other assessments to monitor your health and how you're responding to the study medicine, tebapivat. You would take the study medicine as directed by the research team.

There would also be follow-up appointments after you stop taking the medicine to ensure your well-being. The total length of your participation, including the treatment and follow-up periods, is not specified in this brief summary but would be clearly explained by the study team if you decide to inquire further.

Potential risks and benefits

Participating in this study might offer a potential benefit if tebapivat helps reduce your need for blood transfusions or improves your anaemia. However, like all medicines, tebapivat may have side effects, and some risks are currently unknown as it's a new treatment. The study team will explain any known potential risks and monitor you closely for any unwanted effects. You would always have the right to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (45)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Innovative Clinical Research Institute Whittier
    Verified postcode
    Lakewood, United States
  • David Geffen School of Medicine at UCLA
    Verified postcode
    Los Angeles, United States
  • Emad Ibrahim, MD, Inc.
    Verified postcode
    Redlands, United States
  • Smilow Cancer Hospital at Yale New Haven
    Verified postcode
    New Haven, United States
  • Mayo Clinic Jacksonville - PPDS
    Verified postcode
    Jacksonville, United States
  • Edward H. Kaplan MD & Associates
    Verified postcode
    Skokie, United States
  • Washington University School of Medicine
    Verified postcode
    St Louis, United States
  • Memorial Sloan Kettering Cancer Center
    Verified postcode
    Long Island City, United States
  • Duke Adult Blood and Marrow Clinic
    Verified postcode
    Durham, United States
  • Monash Health, Monash Medical Centre
    Verified postcode
    Clayton, Australia
  • Ordensklinikum Linz GmbH Elisabethinen
    Verified postcode
    Linz, Austria
  • Fakultni nemocnice Ostrava
    Verified postcode
    Ostrava, Czechia

Common questions

What is LR-MDS?

LR-MDS stands for lower-risk myelodysplastic syndromes. It's a condition where your bone marrow doesn't make enough healthy blood cells.

What is tebapivat?

Tebapivat is a new medicine being tested in this study to see if it can help people with LR-MDS make more of their own red blood cells.

What does 'transfusion independence' mean?

'Transfusion independence' means that you no longer need regular blood transfusions to treat your anaemia.

Will I stop my current medications if I join?

The study team will discuss all your current medications with you. If you are taking iron chelation therapy, your dose needs to have been steady for some time before joining.

Who is running this study?

This study is sponsored by a company developing the medicine, and is being conducted by medical professionals at various clinical sites.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Discussion

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