Tebapivat for Sickle Cell Disease

Overseen By Agios Medical Affairs

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Agios Pharmaceuticals, Inc.

Must be taking: Hydroxyurea

Must not be taking: Voxelotor, Crizanlizumab, L-glutamine

Disqualifiers: Chronic transfusion, >10 SCPCs, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

56 Participants Needed

Trial Summary

What is the purpose of this trial?

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 participants with SCD.

Eligibility Criteria

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

I have been diagnosed with a form of sickle cell disease.

My hydroxyurea dose has been stable for 3 months, or I stopped it 3 months ago.

My average hemoglobin levels are between 5.5 and 10.5 g/dL, based on at least two tests.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive tebapivat or placebo orally once daily for 12 weeks in the double-blind period

12 weeks

Open-label extension

Participants who complete the double-blind period may receive tebapivat for up to 52 weeks

Up to 52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

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.

Participant Groups

Group I: Tebapivat 7.5 mg Experimental Treatment 1 Intervention

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.

Group II: Tebapivat 5.0 mg Experimental Treatment 1 Intervention

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.

Group III: Tebapivat 2.5 milligrams (mg) Experimental Treatment 1 Intervention

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.

Group IV: Tebapivat Matched Placebo Placebo Group 2 Interventions

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

Who Is Running the Clinical Trial?

Agios Pharmaceuticals, Inc.

Lead Sponsor

Trials

55

Recruited

4,200+