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)
Status: Recruiting
Location: See all (20) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 35
Healthy Volunteers: f
View:
• Age ≥18 years to ≤35 years for the initial sentinel cohort; for subsequent enrollment patients from ≥12 years up to ≤35 years may be enrolled only upon approval by FDA.
• Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes.
• Severe SCD defined by the occurrence of at least 4 severe VOCs in the 24 months prior to screening despite receiving hydroxyurea or other supportive care measures
Locations
United States
Alabama
University of Alabama at Birmingham
RECRUITING
Birmingham
Arizona
Phoenix Children's Hospital
RECRUITING
Phoenix
California
Lucile Packard Children's Hospital at Standford
RECRUITING
Palo Alto
Florida
Mayo Clinic Florida
RECRUITING
Jacksonville
University of Miami
RECRUITING
Miami
Georgia
Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital
RECRUITING
Atlanta
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Michigan
Henry Ford Cancer Center
RECRUITING
Detroit
Minnesota
University of Minnesota
RECRUITING
Minneapolis
Missouri
Washington University School of Medicine in St. Louis
RECRUITING
St Louis
New Jersey
Hackensack University Medical Center
RECRUITING
Hackensack
New York
Columbia University Irving Medical Center
RECRUITING
New York
Memorial Sloan Kettering Cancer Center
RECRUITING
New York
Ohio
The Cleveland Clinic Foundation
RECRUITING
Cleveland
University Hospitals Cleveland Medical Center
RECRUITING
Cleveland
Pennsylvania
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
South Carolina
Medical University of South Carolina
RECRUITING
Charleston
Tennessee
St Jude Children's Research Hospital
RECRUITING
Memphis
The Children's Hospital at TriStar Centennial
RECRUITING
Nashville
Wisconsin
Medical College of Wisconsin
RECRUITING
Milwaukee
Contact Information
Primary
Medical Information
clinicalinfo@beamtx.com
857-327-8641
Time Frame
Start Date: 2022-08-30
Estimated Completion Date: 2027-02-01
Participants
Target number of participants: 15
Treatments
Experimental: BEAM-101
BEAM-101 manufactured with autologous CD34+ hematopoietic stem cells collected by plerixafor mobilization and edited ex vivo. No maximum dose has been set for BEAM-101; all of the gene edited cells that pass release specifications will be administered to the patient. BEAM 101 will be administered as a single dose by IV infusion.
Related Therapeutic Areas
Sponsors
Leads: Beam Therapeutics Inc.