BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

NCT05456880 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: BTX-AUT-001
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 19

Brief 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

Conditions

Sickle Cell Disease

Keywords

Gene Editing; Sickle Cell; Severe Sickle Cell

Outcome Measures

Primary Outcomes (6)

• Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline

  6 months to time of analysis as compared to baseline

• Proportion of patients with successful neutrophil engraftment

  BEAM-101 administration to month 24

• Time to neutrophil engraftment

  BEAM-101 administration to month 24

• Time to platelet engraftment

  BEAM-101 administration to month 24

• Transplant-related mortality within 100 days after beam-101 treatment

  BEAM-101 administration to day 100

• Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)

  BEAM-101 administration through month 24

Secondary Outcomes (12)

• Proportion of patients experiencing at least 75% reduction in annualized rate of severe VOCs

  Month 6 post BEAM-101 treatment to month 24 as compared to baseline

• Proportion of patients experiencing no severe VOCs

  6 months to time of analysis as compared to baseline

• Change in annualized number of hospitalizations for VOCs

  Month 6 post BEAM-101 treatment to month 24 as compared to baseline

• Change in annualized duration of hospitalizations for VOCs

  Month 6 post BEAM-101 treatment to month 24 as compared to baseline

• Change in RBC transfusions per month and per year for SCD-related indications

  Month 2 post BEAM-101 treatment to month 24 as compared to baseline

Study Arms (1)

BEAM-101

EXPERIMENTAL

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.

Biological: BEAM-101

Interventions

BEAM-101 BIOLOGICAL

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

BEAM-101

Eligibility Criteria

• Age: 12 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age ≥12 years to ≤35 years
• 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

You may not qualify if:

• HbF levels \>20%, obtained at the time of screening on or off hydroxyurea therapy
• Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation
• Available and willing matched sibling donor
• Definitive diagnosis of moyamoya syndrome based on screening brain MRA
• History of overt stroke

Sponsors & Collaborators

• Beam Therapeutics Inc.: lead

Study Sites (19)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Mayo Clinic Florida

Jacksonville, Florida, 32224, United States

Location

University of Miami

Miami, Florida, 33136-1005, United States

Location

Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital

Atlanta, Georgia, 30329, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02215, United States

Location

Henry Ford Cancer Center

Detroit, Michigan, 48202, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Washington University School of Medicine in St. Louis

St Louis, Missouri, 63110, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

The Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

St Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

The Children's Hospital at TriStar Centennial

Nashville, Tennessee, 37203, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (3)

• Ligon JA, Cupit-Link MC, Yu C, Levine J, Foley T, Rotz S, Sharma A, Gomez-Lobo V, Shah NN. Pediatric Cancer Immunotherapy and Potential for Impact on Fertility: A Need for Evidence-Based Guidance. Transplant Cell Ther. 2024 Aug;30(8):737-749. doi: 10.1016/j.jtct.2024.06.006. Epub 2024 Jun 10.

  PMID: 38866240 DERIVED

• Sharma A, Young A, Carroll Y, Darji H, Li Y, Mandrell BN, Nelson MN, Owens CL, Irvine M, Caples M, Jerkins LP, Unguru Y, Hankins JS, Johnson LM. Gene therapy in sickle cell disease: Attitudes and informational needs of patients and caregivers. Pediatr Blood Cancer. 2023 Jun;70(6):e30319. doi: 10.1002/pbc.30319. Epub 2023 Mar 28.

  PMID: 36975201 DERIVED

• Persaud Y, Mandrell BN, Sharma A, Carroll Y, Irvine M, Olufadi Y, Kang G, Hijano DR, Rai P, Hankins JS, Johnson LM. Attitudes toward COVID-19 vaccine among pediatric patients with sickle cell disease and their caregivers. Pediatr Blood Cancer. 2023 May;70(5):e30274. doi: 10.1002/pbc.30274. Epub 2023 Mar 1.

  PMID: 36860093 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 23, 2022
• First Posted: July 13, 2022
• Study Start: August 30, 2022
• Primary Completion (Estimated): February 1, 2028
• Study Completion (Estimated): February 1, 2028
• Last Updated: December 16, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (19)