A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Running, not enrolling · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Sickle Cell Disease

In brief

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.

Key facts

Study ID

NCT05031780

Run by

Agios Pharmaceuticals, Inc.

People needed

286

Starts

2022-02-11

Expected to finish

2030-02-01

Last updated by the study team

2026-06-23

Who can join

Age: 16 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age 16 years or older (18 years or older [France and Germany]); participants age 16 or 17 years must physically have completed puberty;
• 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);
• At least 2 SCPCs and no more than 10 SCPCs in the past 12 months;
• Hemoglobin at least 5.5 and 10.5 gram per deciliter (g/dL) at the most. Hemoglobin concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period;
• If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before starting study drug. Discontinuation of hydroxyurea requires a 90-day washout prior to informed assent/consent;
• Women capable of becoming pregnant must agree to use 2 forms of contraception.

You may not qualify if…

• Pregnant, breastfeeding, or parturient;
• Receiving regularly scheduled transfusions;
• Hepatobiliary disorders including but not limited to significant liver disease or gallbladder disease;
• Severe kidney disease;
• Prior exposure to gene therapy or prior bone marrow or stem cell transplantation;
• Currently receiving treatment with a disease-modifying therapy for SCD (eg, voxelotor, crizanlizumab, L-glutamine), with the exception of hydroxyurea. The last dose of voxelotor, crizanlizumab, and L-glutamine must have been administered at least 90 days before randomization;
• Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered at least 90 days before starting study drug;
• Received treatment on another investigational trial within 90 days prior to start of study drug or plans to participate in another investigational drug trial;
• Taking medications that are strong inhibitors of CYP3A4/5 or strong inducers of CYP3A4 that cannot be stopped in an acceptable timeframe before starting study drug (timeframe will be discussed with your doctor).

Where it is running

• University of California San Diego — La Jolla, California, United States
• UCLA Health — Los Angeles, California, United States
• Children's Hospital Oakland — Oakland, California, United States
• University of Connecticut Health Center — Farmington, Connecticut, United States
• Children's National Hospital — Washington D.C., District of Columbia, United States
• MedStar Washington Hospital Center — Washington D.C., District of Columbia, United States
• Sylvester Comprehensive Cancer Center-Miami — Miami, Florida, United States
• University of Chicago Medical Center — Chicago, Illinois, United States
• Riley Hospital For Children — Indianapolis, Indiana, United States
• LSU Health Sciences Center - Shreveport — Shreveport, Louisiana, United States
• Kondele Children's Hospital — Kisumu, Kenya
• Kaiser Permanente - Largo Medical Center — Largo, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Boston Medical Center & Boston University School of Medicine — Boston, Massachusetts, United States
• University of Michigan — Ann Arbor, Michigan, United States
• Children's Hospital of Michigan — Detroit, Michigan, United States
• Southern Specialty Clinic — Flowood, Mississippi, United States
• Mississippi Center for Advanced Medicine — Madison, Mississippi, United States
• Cure 4 The Kids Foundation, A Division of Roseman University of Health Sciences — Las Vegas, Nevada, United States
• East Carolina University - Brody School of Medicine — Greenville, North Carolina, United States
• Penn Medicine - University of Pennsylvania Health System — Philadelphia, Pennsylvania, United States
• St. Christopher's Hospital for Children — Philadelphia, Pennsylvania, United States
• Lifespan at Rhode Island Hospital — Providence, Rhode Island, United States
• National Heart Lung and Blood Institute — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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