Mitapivat for Sickle Cell Disease

Recruiting at 105 trial locations

Overseen By Agios Medical Affairs

Age: Any Age

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Agios Pharmaceuticals, Inc.

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

Compensation: Varies

Number of Visits: Unknown

286 Participants Needed

Trial Summary

What is the purpose of this trial?

This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of hemoglobin in the blood and to reduce or prevent the occurrence of sickle cell pain crises. In addition, the long-term effect of mitapivat on efficacy and safety will be explored in an open-label extension portion.

Eligibility Criteria

Adults and teens aged 16 or older with sickle cell disease (SCD) who've had 2-10 pain crises in the past year can join. They need stable hemoglobin levels between 5.5 and 10.5 g/dL, possibly on a steady dose of hydroxyurea for at least three months, and must use two forms of contraception if they can have children. People with severe liver, gallbladder, kidney diseases, prior gene therapy or transplants, recent other SCD treatments except hydroxyurea, or certain drug interactions are excluded.

Inclusion Criteria

My hydroxyurea dose has been the same for the last 90 days.

I am 16 or older and have completed puberty, or I am 18 or older.

I have been diagnosed with sickle cell disease.

Exclusion Criteria

I am not on strong CYP3A4/5 medications that can't be stopped before starting the study drug.

I do not have significant liver or gallbladder disease.

Pregnant, breastfeeding, or parturient

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 2 Treatment

Participants receive mitapivat 50 mg or 100 mg BID or placebo for 12 weeks

12 weeks

Phase 3 Treatment

Participants receive mitapivat 100 mg BID or placebo for 52 weeks

52 weeks

Open-label Extension

Participants may opt into receiving mitapivat 50 mg or 100 mg BID for 216 weeks

216 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Mitapivat
• Mitapivat-matching placebo

Trial Overview The trial is testing Mitapivat against a placebo to see if it raises hemoglobin levels and reduces sickle cell pain crises. Participants will be randomly assigned to receive either Mitapivat or a matching dummy pill (placebo). The study includes an initial phase to set the right dose followed by a longer-term evaluation of its effects.

Participant Groups

Group I: Phase 3: Open-Label Extension Period Experimental Treatment 1 Intervention

Participants may choose to receive mitapivat 100 mg BID for 216 weeks after the Double-blind Period. Participants who received mitapivat-matching placebo in the double-blind period, may choose to receive mitapivat 100 mg BID for 216 weeks after the Double-blind Period.

Group II: Phase 3: Mitapivat 100 mg BID Experimental Treatment 1 Intervention

Double-blind Period: Mitapivat 100 mg BID for 52 weeks.

Group III: Phase 2: Open-Label Extension Period Experimental Treatment 1 Intervention

Participants who received mitapivat 50mg BID in the double-blind period may choose to receive mitapivat 50mg BID for 216 weeks after. Participants who received mitapivat 100mg BID in the double-blind period may choose to receive mitapivat 100 mg BID for 216 weeks after. Participants who received mitapivat-matching placebo in the double-blind period, may be randomized to receive either mitapivat 50 mg or 100 mg BID for 216 weeks after.

Group IV: Phase 2: Mitapivat 50 mg BID Experimental Treatment 1 Intervention

Double-blind Period: Mitapivat 50 milligrams (mg) twice daily (BID) for 12 weeks.

Group V: Phase 2: Mitapivat 100 mg BID Experimental Treatment 1 Intervention

Double-blind Period: Mitapivat 100 mg BID for 12 weeks.

Group VI: Phase 2: Placebo Placebo Group 1 Intervention

Double-blind Period: Mitapivat-matching placebo for 12 weeks.

Group VII: Phase 3: Placebo Placebo Group 1 Intervention

Double-blind Period: Mitapivat-matching placebo for 52 weeks.

Who Is Running the Clinical Trial?

Agios Pharmaceuticals, Inc.

Lead Sponsor

Trials

55

Recruited

4,200+