Mitapivat for Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

+105 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: Agios Pharmaceuticals, Inc.

Prior Safety Data

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.

+3 more

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

+6 more

Participant Groups

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.

7Treatment groups

Experimental Treatment

Placebo Group

Group I: Phase 3: Open-Label Extension PeriodExperimental Treatment1 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 BIDExperimental Treatment1 Intervention

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

Group III: Phase 2: Open-Label Extension PeriodExperimental Treatment1 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 BIDExperimental Treatment1 Intervention

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

Group V: Phase 2: Mitapivat 100 mg BIDExperimental Treatment1 Intervention

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

Group VI: Phase 2: PlaceboPlacebo Group1 Intervention

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

Group VII: Phase 3: PlaceboPlacebo Group1 Intervention

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