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
Trial Overview
The study aims to see if Tebapivat can improve anemia in SCD patients compared to a placebo. It will also look for the best dose by observing changes in hemoglobin levels after treatment.
How Is the Trial Designed?
4
Treatment groups
Experimental Treatment
Placebo Group
Participants will receive 7.5 mg tebapivat orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be eligible to receive the same dose in the OLE period for up to 52 weeks.
Participants will receive 5.0 mg tebapivat orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be eligible to receive the same dose in the OLE period for up to 52 weeks.
Participants will receive 2.5 mg tebapivat orally, once daily (QD) for 12-weeks in the double-blind (DB) period. Participants who complete the DB Period will be eligible to receive the same dose in the Open-Label Extension (OLE) period for up to 52 weeks.
Participants will receive a matched placebo, orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be randomized in 1:1:1 to receive tebapivat 2.5 mg QD, tebapivat 5.0 mg QD, or tebapivat 7.5 mg QD in the OLE period for up to 52 weeks
Find a Clinic Near You
Who Is Running the Clinical Trial?
Agios Pharmaceuticals, Inc.
Lead Sponsor
Citations
Safety and efficacy of mitapivat in sickle cell disease (RISE ...
Both treatment groups showed a statistically significant haemoglobin response rate versus placebo (12 [46%] of 26 patients in the mitapivat 50 ...
2.
ashpublications.org
ashpublications.org/bloodrci/article/1/2/100014/546279/Long-term-mitapivat-treatment-is-safe-andLong-term mitapivat treatment is safe and efficacious in ...
Median of 2.53-year follow-up of mitapivat showed favorable safety and tolerability in patients with SCD. Sustained improvements in Hb, ...
Agios EHA 2025 Data Room
In the study, mitapivat showed sustained efficacy and tolerability over three years, including improvements in anemia, hemolysis, painful vaso-occlusive crises ...
Long-term mitapivat treatment is safe and efficacious in ...
Median of 2.53-year follow-up of mitapivat showed favorable safety and tolerability in patients with SCD. · Sustained improvements in Hb, hemolytic markers, and ...
5.
hematologyadvisor.com
hematologyadvisor.com/news/mitapivat-sickle-cell-disease-scd-phase-2-results-support-investigation/Phase 2 Results Support Continued Investigation Into ...
Hemoglobin response rates were 46% for the mitapivat 50-mg group, 50% for the mitapivat 100-mg group, and 4% in the placebo group (2-sided P =.
6.
ashpublications.org
ashpublications.org/blood/article/144/Supplement%201/2496/533222/Results-from-a-Phase-1-Study-to-Assess-the-SafetyResults from a Phase 1 Study to Assess the Safety, Tolerability ...
Aims: To identify a well-tolerated and pharmacologically active dose of tebapivat in pts with SCD. Methods: Two dose levels of tebapivat were ...
NCT06924970 | A Dose-Finding Study of Tebapivat to ...
The main purpose of this study is to compare the effect of tebapivat versus placebo on anemia and to detect a dose-response for hemoglobin (Hb) response in ...
8.
investor.agios.com
investor.agios.com/news-releases/news-release-details/agios-present-new-data-mitapivat-and-tebapivat-rare-bloodAgios to Present New Data on Mitapivat and Tebapivat in ...
Sickle Cell Disease. Results From A Phase 1 Study To Assess The Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of Tebapivat (AG ...
Results from a Phase 1 Study to Assess the Safety ...
Tebapivat (formerly AG-946) is an oral, potent, allosteric pyruvate kinase activator that is under clinical investigation as a potential therapy for rare ...
Tebapivat Improves Red Cell Deformability and Decreases ...
In conclusion, tebapivat treatment was associated with improved RBC deformability and reduced sickling in patients with SCD. These effects are ...
Other People Viewed
By Subject
By Trial
Related Searches
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.