Tebapivat for Sickle Cell Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called tebapivat for individuals with sickle cell disease (SCD). The main goal is to determine if tebapivat can improve anemia by increasing hemoglobin levels compared to a placebo. Participants will receive different doses of tebapivat to identify the most effective one. Suitable candidates for this trial include those with a confirmed diagnosis of SCD who do not require regular blood transfusions. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of participants.
Will I have to stop taking my current medications?
If you are taking hydroxyurea, you must have been on a stable dose for at least 90 days before joining the trial. If you need to stop hydroxyurea, you must wait 90 days before joining. Other medications like voxelotor, crizanlizumab, or L-glutamine must be stopped 90 days before joining.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that tebapivat appears promising based on early safety studies. In one study, researchers tested two different doses of tebapivat in people with sickle cell disease (SCD). The results indicated that participants tolerated the treatment well, with no major safety issues reported.
Another study found that tebapivat increased the flexibility of red blood cells and reduced cell sickling in people with SCD. This suggests the treatment not only seems safe but might also improve the condition.
As this is a Phase 2 trial, the treatment has already passed initial safety tests in earlier studies. This phase focuses more on the treatment's effectiveness while still monitoring safety.12345Why are researchers excited about this trial's treatments?
Tebapivat is unique because it specifically targets the underlying issues in sickle cell disease by enhancing red blood cell function. Unlike standard treatments like hydroxyurea and blood transfusions, which mainly manage symptoms and complications, tebapivat works by activating pyruvate kinase-R. This activation helps improve red blood cell energy metabolism, potentially reducing sickling and increasing red blood cell lifespan. Researchers are excited about tebapivat because it offers a new approach by addressing the disease at a cellular level, which could lead to more effective management of sickle cell disease with fewer side effects.
What evidence suggests that this trial's treatments could be effective for sickle cell disease?
Research has shown that tebapivat, also known as mitapivat, may help treat sickle cell disease (SCD). Studies have found that tebapivat can significantly raise hemoglobin levels in patients, improving anemia—a common issue in SCD. One study found that about 46% of patients taking a similar drug, mitapivat, experienced a positive increase in hemoglobin compared to only 4% in the placebo group. Long-term use of mitapivat has proven safe, with ongoing benefits such as fewer painful episodes and improved blood health. Overall, tebapivat boosts energy production in red blood cells, potentially reducing symptoms of sickle cell disease.
In this trial, participants will receive different doses of tebapivat or a matched placebo to further evaluate its effectiveness and safety.678910Are You a Good Fit for This Trial?
This trial is for people with Sickle Cell Disease (SCD) who have a confirmed diagnosis and hemoglobin levels between 5.5 and 10.5 g/dL, based on multiple tests. Participants must have stable hydroxyurea doses for at least 90 days or have stopped it for the same duration before consenting.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive tebapivat or placebo orally once daily for 12 weeks in the double-blind period
Open-label extension
Participants who complete the double-blind period may receive tebapivat for up to 52 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Tebapivat
Find a Clinic Near You
Who Is Running the Clinical Trial?
Agios Pharmaceuticals, Inc.
Lead Sponsor