Active not recruitingPHASE2INTERVENTIONAL

A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)

This study is investigating a new medication called tebapivat for people with sickle cell disease. The main goal is to understand how well tebapivat works to improve anaemia, which means having too few red blood cells. Researchers are also looking to see if certain doses of tebapivat are more effective than others. To do this, some participants will receive tebapivat, while others will receive a 'dummy' medicine, called a placebo, which looks the same but contains no active drug. This comparison helps researchers understand if any improvements are truly due to tebapivat. The study also aims to check the safety of tebapivat and any side effects it might have.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Agios Pharmaceuticals, Inc.

Enrolment target

Start

01 May 2025

Estimated completion

01 May 2027

Sickle Cell Disease

What is this study about?

Sickle cell disease is a lifelong condition that affects your red blood cells. These cells, which are normally round and flexible, become stiff and C-shaped, like a sickle. This can cause various health problems, including anaemia (when your body doesn't produce enough healthy red blood cells) and episodes of severe pain, often called sickle cell pain crises.

This research study is looking at a new potential medicine called tebapivat. The main aim is to see if tebapivat can help improve anaemia in people with sickle cell disease. Researchers will also be comparing different doses of tebapivat to find out which dose might work best. To make sure the results are clear, some people in the study will receive tebapivat, while others will receive a 'placebo', which is an inactive substance that looks just like the real medicine but contains no active drug. This helps the researchers determine if any changes are truly due to the study medicine.

By taking part in this study, you could help doctors learn more about tebapivat and whether it could be a helpful treatment option for people living with sickle cell disease in the future. The study will also carefully monitor any side effects to understand how safe tebapivat is.

Key takeaways

Tests a new medicine, tebapivat, for sickle cell disease.
Aims to improve anaemia and check safety.
Compares tebapivat to a 'dummy' medicine (placebo).
Suitable for people aged 16 and over with confirmed sickle cell disease.
Close medical monitoring throughout the study.
Participation is voluntary, and you can withdraw anytime.

Who may be eligible?

To join this study, you need to have a confirmed diagnosis of sickle cell disease. Your haemoglobin levels (which measure the amount of red blood cells in your blood) need to be within a specific range, usually between 5.5 and 10.5 g/dL, based on recent blood tests.

If you are currently taking a medicine called hydroxyurea, your dose must have been stable for at least three months. If you recently stopped taking hydroxyurea, you'd need to wait three months before joining. You must be at least 16 years old.

There are also some reasons why you might not be able to join. For example, if you regularly have blood transfusions, you won't be able to participate. However, if you only have occasional transfusions for anaemia or pain crises, you might still be eligible, provided you haven't had one within the last 60 days. You also can't have had more than 10 sickle cell pain crises in the past year, or been in hospital for a crisis within two weeks of the study starting. If you're on certain other sickle cell medications, or some steroids, you might need to stop them for a period before joining.

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.

Do you have a confirmed diagnosis of sickle cell disease (e.g., HbSS, HbSC)?
Are your haemoglobin levels between 5.5 and 10.5 g/dL?
If you take hydroxyurea, has your dose been stable for at least 3 months?
Have you not had more than 10 sickle cell pain crises in the last year?
Have you avoided regular blood transfusions?
Are you 16 years old or older?

Answer every question to see your result.

What does participation involve?

Information about how taking part will specifically involve you is not fully detailed in the provided summary. However, generally, taking part in a study like this would typically involve:

* **Screening tests:** This would include various medical checks, blood tests, and reviewing your medical history to make sure you're suitable for the study. * **Taking the study medicine:** You would take either tebapivat or the placebo, likely in pill form, for a set period. * **Regular visits:** You would have scheduled appointments at the clinic for check-ups, blood tests, and to discuss any side effects you might be experiencing. * **Monitoring:** The research team would closely monitor your health, especially your anaemia levels and any sickle cell pain crises you might have. * **Follow-up:** After you stop taking the study medicine, there would likely be follow-up visits or calls to ensure your well-being.

Potential risks and benefits

Participating in this study might offer some potential benefits, such as closely monitored medical care and access to a new medicine that could potentially improve your anaemia if it proves to be effective. However, there are also potential risks; tebapivat might cause side effects, some of which could be unexpected or unpleasant. Since you might receive a placebo, there's a chance your health condition might not improve during the study. All study participants have the right to withdraw from the study at any time and for any reason, without affecting their standard medical care.

Locations (30)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

UCHealth at University of Colorado Anschutz Medical Campus
Verified postcode
Aurora, United States
UConn Health
Verified postcode
Farmington, United States
Children's National Hospital
Verified postcode
Washington D.C., United States
MedStar Washington Hospital Center
Verified postcode
Washington D.C., United States
Emory-Children's Center/ Children's Healthcare of Atlanta: Arthur M. Blank Hospital
Verified postcode
Atlanta, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Verified postcode
Chicago, United States
Boston Medical Center & Boston University School of Medicine
Verified postcode
Boston, United States
Henry Ford Health System
Verified postcode
Detroit, United States
Children's Hospital of Michigan
Verified postcode
Detroit, United States
Icahn School of Medicine at Mt. Sinai
Verified postcode
New York, United States
Montefiore Medical Center
Verified postcode
The Bronx, United States
Thomas Jefferson University Hospital
Verified postcode
Philadelphia, United States

Common questions

What is tebapivat?

Tebapivat is a new investigational medicine being tested to see if it can help improve anaemia in people with sickle cell disease.

Why is there a placebo?

A placebo is a 'dummy' medicine with no active drug. It helps researchers understand if any changes in your health are truly caused by tebapivat, rather than other factors.

How long will the study last?

The information provided doesn't specify the exact duration, but clinical trials usually involve a period of taking the medicine, followed by monitoring.

Will I know if I'm getting tebapivat or the placebo?

No, in this type of study, neither you nor your doctor will typically know whether you are receiving tebapivat or the placebo until after the study is over. This is to ensure unbiased results.

What should I do if I feel worse during the study?

You should always tell the study team immediately if you experience any new or worsening symptoms or feel unwell while taking part in the study.