Before you are fully registered as a participant in RISE UP, a medical team will assess your medical history and health to ensure you can participate. See Deciding to Enroll to get a better idea if the trial is right for you.

Phase 2 of the global RISE UP study showed a statistically significant increase in hemoglobin response rate over 12 weeks with mitapivat when compared to placebo.

A total of 79 patients were registered in the global RISE UP phase 2 study. While 27 patients received placebo, there were 52 patients that received mitapivat.

Mitapivat improved the markers of hemolysis and erythropoiesis compared to placebo. Markers for hemolysis tell you how healthy your red blood cells are, and markers for erythropoiesis tell you the speed at which your red blood cells and other blood cells are being produced.

Mitapivat also showed a more than 50% reduction in sickle cell pain crises (SCPC) compared to placebo.

Yes, the same providers used in phase 2 will be used for phase 3, as well as some additional providers.

Mitapivat has been studied since March 2014.

Over 700 patients, including healthy volunteers, have been treated with mitapivat for various diseases.

Mitapivat has been studied for over 5 years.

Mitapivat as a disease-modifying therapy may improve your disease, but will not alter your DNA.

During phase 3 of the study, for every three patients participating, two will be on mitapivat and one will be on placebo.

However, there is a 4-year optional open-label extension study period where everyone receives mitapivat.

The purpose of this phase 3 study is to determine if mitapivat is safe and effective.

No patients on mitapivat or placebo discontinued treatment due to side effects during the phase 2 study.

Across all studies of mitapivat, side effects seen in 10% or more patients included headache, feeling tired, nausea, joint pain, and COVID-19.

Mitapivat may affect the way other medications work, and other medications may affect the way mitapivat works. Taking mitapivat with other medications may also cause side effects.

Talk to your doctor about the medications you currently take (including prescription medications, over-the-counter medications and supplements, and any product containing grapefruit). Your study doctor will let you know which medications are safe to continue taking while on mitapivat.

A placebo is a pill that looks exactly like the study medication but does not contain any medicine. Placebos provide a way to compare the results produced by the investigational medicine. “Double-blind” means that neither you nor your doctor will know whether you’re taking placebo or the study medication, so as not to influence the results or evaluations.

Throughout the study, your name and all your personal medical information will be kept completely confidential. Each participant in the study will be assigned a unique identifier. Records and data collected throughout the study will not contain your name or personal identifying information, but rather the unique identifier.

You have other things happening in your life, and battling sickle cell disease every day is a huge challenge. If you need help during the study, support may be available for:

• Caregivers
• Childcare
• Travel
• Transportation
• Stipends
• Nurses for remote visits
• Virtual visits

The above services depend on local and national regulations. Please check with your local investigator to confirm which services are available in your area. More information about support will be available on your specific enrollment website.