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BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
Sponsor: Beam Therapeutics Inc.
Summary
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
Official title: 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)
Key Details
Gender
All
Age Range
12 Years - 35 Years
Study Type
INTERVENTIONAL
Enrollment
15
Start Date
2022-08-30
Completion Date
2028-02
Last Updated
2025-12-16
Healthy Volunteers
No
Conditions
Interventions
BEAM-101
Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan
Locations (19)
University of Alabama at Birmingham
Birmingham, Alabama, United States
Phoenix Children's Hospital
Phoenix, Arizona, United States
Mayo Clinic Florida
Jacksonville, Florida, United States
University of Miami
Miami, Florida, United States
Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital
Atlanta, Georgia, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Henry Ford Cancer Center
Detroit, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Columbia University Irving Medical Center
New York, New York, United States
Memorial Sloan Kettering Cancer Center
New York, New York, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
The Cleveland Clinic Foundation
Cleveland, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
St Jude Children's Research Hospital
Memphis, Tennessee, United States
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States