A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) (NCT05031780)
A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of California San Diego — La Jolla, California
Age range: 16 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (91 Sites)
Showing the first 25 of 91 sites.
- University of California San Diego — La Jolla, California
- UCLA Health — Los Angeles, California
- Children's Hospital Oakland — Oakland, California
- University of Connecticut Health Center — Farmington, Connecticut
- Children's National Hospital — Washington D.C., District of Columbia
- MedStar Washington Hospital Center — Washington D.C., District of Columbia
- Sylvester Comprehensive Cancer Center-Miami — Miami, Florida
- University of Chicago Medical Center — Chicago, Illinois
- Riley Hospital For Children — Indianapolis, Indiana
- LSU Health Sciences Center - Shreveport — Shreveport, Louisiana
- National Heart Lung and Blood Institute — Bethesda, Maryland
- Kaiser Permanente - Largo Medical Center — Largo, Maryland
- Massachusetts General Hospital — Boston, Massachusetts
- Boston Children's Hospital — Boston, Massachusetts
- Boston Medical Center & Boston University School of Medicine — Boston, Massachusetts
- University of Michigan — Ann Arbor, Michigan
- Children's Hospital of Michigan — Detroit, Michigan
- Southern Specialty Clinic — Flowood, Mississippi
- Mississippi Center for Advanced Medicine — Madison, Mississippi
- Cure 4 The Kids Foundation, A Division of Roseman University of Health Sciences — Las Vegas, Nevada
- East Carolina University - Brody School of Medicine — Greenville, North Carolina
- Penn Medicine - University of Pennsylvania Health System — Philadelphia, Pennsylvania
- St. Christopher's Hospital for Children — Philadelphia, Pennsylvania
- Lifespan at Rhode Island Hospital — Providence, Rhode Island
- University of Texas Health Science Center of Houston — Houston, Texas
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Sickle Cell Disease
- Kidney Function in Sickle Cell Anemia — Chapel Hill, North Carolina
- BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease — Birmingham, Alabama
- Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease — Bethesda, Maryland
- Acupressure in Patients With Sickle Cell Disease — Irvine, California
- Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC) — Chicago, Illinois
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) — Charlotte, North Carolina
Explore by condition: All Sickle Cell Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.