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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)

Study 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

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Interventions

BEAM-101BIOLOGICAL
Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan

Study Locations

FacilityCityStateCountry
University of Alabama at BirminghamBirminghamAlabamaUnited States
Phoenix Children's HospitalPhoenixArizonaUnited States
Mayo Clinic FloridaJacksonvilleFloridaUnited States
University of MiamiMiamiFloridaUnited States
Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston HospitalAtlantaGeorgiaUnited States
Boston Children's HospitalBostonMassachusettsUnited States
Henry Ford Cancer CenterDetroitMichiganUnited States
University of MinnesotaMinneapolisMinnesotaUnited States
Washington University School of Medicine in St. LouisSt LouisMissouriUnited States
Hackensack University Medical CenterHackensackNew JerseyUnited States
Columbia University Irving Medical CenterNew YorkNew YorkUnited States
Memorial Sloan Kettering Cancer CenterNew YorkNew YorkUnited States
University Hospitals Cleveland Medical CenterClevelandOhioUnited States
The Cleveland Clinic FoundationClevelandOhioUnited States
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvaniaUnited States
Medical University of South CarolinaCharlestonSouth CarolinaUnited States
St Jude Children's Research HospitalMemphisTennesseeUnited States
The Children's Hospital at TriStar CentennialNashvilleTennesseeUnited States
Medical College of WisconsinMilwaukeeWisconsinUnited States

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026