Long-Term Mitapivat for Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

Overseen byJulia Z Xu, M.D.

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

No Placebo Group

Trial Summary

What is the purpose of this trial?Background: Sickle cell disease (SCD) is a disorder that causes episodes of acute pain and progressive organ damage. Ways to manage SCD have evolved slowly. Treatments do not always work. Researchers want to see if a drug called mitapivat can help people with SCD. Objective: To test the long-term tolerability and safety of mitapivat (or AG-348) in people with SCD. Eligibility: Adults age 18-70 with SCD who took part in and benefited from NIH study #19H0097. Design: Participants will be screened with a medical history and physical exam. They will give a blood sample. They will have an electrocardiogram to test heart function. Participants will repeat some of the screening tests during the study. Participants will complete 6-minute walk tests to measure mobility and function. They will have transthoracic echocardiograms to measure heart and lung function. They will have dual-energy X-ray absorptiometry scans to measure bone health. They will complete online questionnaires that measure their overall health and well-being. Participants will take the study drug in the form of a tablet twice a day. Participants will keep a study diary. They will record any symptoms they may have. Participation will last for about 54 weeks. After 48 weeks, participants can either keep taking the study drug for 48 more weeks or be tapered off of the study drug to complete the study. Those who are on the study for 1 year will have 10 study visits. Those who are on the study for 2 years will have 14 study visits.

Eligibility Criteria

Adults aged 18-70 with Sickle Cell Disease (SCD) who previously participated in NIH study #19H0097 and benefited from it. They must have stable organ function, no recent blood transfusions, and for women of childbearing potential, a commitment to use two forms of contraception. Exclusions include significant heart rhythm issues, uncontrolled medical conditions like hypertension or diabetes, active infections or certain drug allergies.

Inclusion Criteria

I am a woman who can have children and have a negative pregnancy test.

My blood clotting tests are within normal limits, unless I'm on blood thinners.

My organs are functioning well.

+13 more

Exclusion Criteria

2.3 Have a significant medical condition that confers an unacceptable risk to participating in the study, and/or that could confound the interpretation of the study data. Such significant medical conditions include, but are not limited to the following:

I have had gallstones or gallbladder inflammation but it's treated now.

My high blood pressure is not controlled by medication.

+22 more

Participant Groups

The trial is testing the long-term safety and effectiveness of mitapivat tablets taken twice daily by people with SCD. It extends a previous study to see if benefits continue or improve over time. Participants will undergo various health assessments including physical exams, blood tests, heart checks (ECG), mobility tests (6-minute walk), echocardiograms for heart/lung function, bone scans (DXA), and health questionnaires.

1Treatment groups

Experimental Treatment

Group I: 1Experimental Treatment1 Intervention

Subjects will be treated with a maintenance dose of mitapivat previously assessed for safety and tolerability in the Phase I study for an initial 48 weeks and undergo safety monitoring, evaluation of pharmacokinetics and pharmacodynamics, and assessment of secondary laboratory and clinical endpoints at pre-specified intervals during the study period.