🏛️ Verified NIH Record • 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)

🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic Sponsor: Beam Therapeutics Inc.

🩺 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 — Birmingham, Alabama

Age range: 12 Years to 35 Years

🧪 Interventions in This Study

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria ↗

📍 All Participating Trial Locations (19 Sites)

  • University of Alabama at Birmingham — Birmingham, Alabama
  • Phoenix Children's Hospital — Phoenix, Arizona
  • Mayo Clinic Florida — Jacksonville, Florida
  • University of Miami — Miami, Florida
  • Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital — Atlanta, Georgia
  • Boston Children's Hospital — Boston, Massachusetts
  • Henry Ford Cancer Center — Detroit, Michigan
  • University of Minnesota — Minneapolis, Minnesota
  • Washington University School of Medicine in St. Louis — St Louis, Missouri
  • Hackensack University Medical Center — Hackensack, New Jersey
  • Columbia University Irving Medical Center — New York, New York
  • Memorial Sloan Kettering Cancer Center — New York, New York
  • University Hospitals Cleveland Medical Center — Cleveland, Ohio
  • The Cleveland Clinic Foundation — Cleveland, Ohio
  • Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
  • Medical University of South Carolina — Charleston, South Carolina
  • St Jude Children's Research Hospital — Memphis, Tennessee
  • The Children's Hospital at TriStar Centennial — Nashville, Tennessee
  • Medical College of Wisconsin — Milwaukee, Wisconsin

💰 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.

💡 How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Review the official NIH record below to find the coordinating site.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
🏛️ View Official NIH Record ↗