๐๏ธ Verified NIH Record โข Updated 2025-12-15
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease (NCT05456880)
A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients With Sickle Cell Disease and Severe Vaso-Occlusive Crises (Beacon Trial)
๐ต Active, Not Recruiting Phase1 / Phase2 ๐ Investigational Drug / Biologic Sponsor: Beam Therapeutics Inc.
๐ฉบ Plain-English Summary (8th-Grade Level)
This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD
Who can join: Review the eligibility criteria below.
Location: University of Alabama at Birmingham โ Birmingham, Alabama
Age range: 12 Years to 35 Years
๐งช Interventions in This Study
๐ "Do I Qualify?" โ 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria โ
๐ All Participating Trial Locations (19 Sites)
- University of Alabama at Birmingham โ Birmingham, Alabama
- Phoenix Children's Hospital โ Phoenix, Arizona
- Mayo Clinic Florida โ Jacksonville, Florida
- University of Miami โ Miami, Florida
- Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital โ Atlanta, Georgia
- Boston Children's Hospital โ Boston, Massachusetts
- Henry Ford Cancer Center โ Detroit, Michigan
- University of Minnesota โ Minneapolis, Minnesota
- Washington University School of Medicine in St. Louis โ St Louis, Missouri
- Hackensack University Medical Center โ Hackensack, New Jersey
- Columbia University Irving Medical Center โ New York, New York
- Memorial Sloan Kettering Cancer Center โ New York, New York
- University Hospitals Cleveland Medical Center โ Cleveland, Ohio
- The Cleveland Clinic Foundation โ Cleveland, Ohio
- Children's Hospital of Philadelphia โ Philadelphia, Pennsylvania
- Medical University of South Carolina โ Charleston, South Carolina
- St Jude Children's Research Hospital โ Memphis, Tennessee
- The Children's Hospital at TriStar Centennial โ Nashville, Tennessee
- Medical College of Wisconsin โ Milwaukee, Wisconsin
๐ฐ 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 ยง 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 โ Chapel Hill, North Carolina
- Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease โ Bethesda, Maryland
- Acupressure in Patients With Sickle Cell Disease โ Irvine, California
- Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC) โ Chicago, Illinois
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) โ Charlotte, North Carolina
Explore by condition: All Sickle Cell Disease clinical trials
๐ก 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.