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Allosteric Activator

Mitapivat for Sickle Cell Disease

Phase 2 & 3

Recruiting

Research Sponsored by Agios Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before starting study drug

Documented diagnosis of sickle cell disease (SCD) (HbSS, HbSC [combined heterozygosity for hemoglobins S and C], HbS/beta 0- thalassemia, HbS/ beta plus thalassemia, or other sickle cell syndrome variants)

Must not have

Prior exposure to gene therapy or prior bone marrow or stem cell transplantation

Receiving regularly scheduled transfusions

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial will test how well mitapivat works compared to placebo in treating sickle cell disease by increasing hemoglobin levels and reducing or preventing sickle cell pain crises. The long-term effect of mitapivat will also be explored.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Possible side effects include but aren't limited to headaches, nausea, fatigue, insomnia and muscle pains. Since this is part of what the study is investigating; all potential side effects may not be known yet.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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I have been diagnosed with sickle cell disease.

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I've had 2 to 10 sickle cell pain crises in the last year.

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I agree to use two forms of birth control.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had gene therapy or a bone marrow/stem cell transplant before.

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I regularly receive blood transfusions.

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I have severe kidney disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug