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Active, not recruitingNCT05456880BEACONUpdated Dec 16, 2025

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

A Phase 1/2 interventional study of BEAM-101 in Sickle Cell Disease, sponsored by Beam Therapeutics Inc.. Active, not recruiting at 19 sites in United States. Open to participants aged 12 Years to 35 Years. Per ClinicalTrials.gov, last updated 2025-12-16.

Sponsored by Beam Therapeutics Inc. · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
15
Allocation
Not applicable
Ages
12 Years to 35 Years
Sex
All
01

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

02

Conditions studied

  • Sickle Cell Disease

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Keywords

  • Gene Editing
  • Sickle Cell
  • Severe Sickle Cell
03

Who can participate

Ages eligible
12 Years to 35 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria Include:

  1. Age ≥12 years to ≤35 years
  2. Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes.
  3. 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

Key Exclusion Criteria Include:

  1. HbF levels >20%, obtained at the time of screening on or off hydroxyurea therapy
  2. Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation
  3. Available and willing matched sibling donor
  4. Definitive diagnosis of moyamoya syndrome based on screening brain MRA
  5. History of overt stroke
04

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
15 participants (estimated)

Study arms

  • 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.

    Biological: BEAM-101

Interventions

  • BiologicalBEAM-101

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

05

What researchers measure

Primary outcomes

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

    Time frame: 6 months to time of analysis as compared to baseline

  2. Proportion of patients with successful neutrophil engraftment

    Time frame: BEAM-101 administration to month 24

  3. Time to neutrophil engraftment

    Time frame: BEAM-101 administration to month 24

  4. Time to platelet engraftment

    Time frame: BEAM-101 administration to month 24

  5. Transplant-related mortality within 100 days after beam-101 treatment

    Time frame: BEAM-101 administration to day 100

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

    Time frame: BEAM-101 administration through month 24

Secondary outcomes

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

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

  2. Proportion of patients experiencing no severe VOCs

    Time frame: 6 months to time of analysis as compared to baseline

  3. Change in annualized number of hospitalizations for VOCs

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

  4. Change in annualized duration of hospitalizations for VOCs

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

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

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

  6. Change in total Hgb (g/dL) concentration over time

    Time frame: Baseline to month 24

  7. Proportion of patients with HbF ≥30%, for at least 3 months

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

  8. Change in lactate dehydrogenase (LDH) over time

    Time frame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline

  9. Change in total bilirubin over time

    Time frame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline

  10. Change in free Hgb over time

    Time frame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline

  11. Change in haptoglobin over time

    Time frame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline

  12. Change in reticulocyte count over time

    Time frame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline

06

Study locations

19 sites
  • University of Alabama at Birmingham
    Birmingham, Alabama 35233, United States
  • Phoenix Children's Hospital
    Phoenix, Arizona 85016, United States
  • Mayo Clinic Florida
    Jacksonville, Florida 32224, United States
  • University of Miami
    Miami, Florida 33136-1005, United States
  • Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital
    Atlanta, Georgia 30329, United States
  • Boston Children's Hospital
    Boston, Massachusetts 02215, United States
  • Henry Ford Cancer Center
    Detroit, Michigan 48202, United States
  • University of Minnesota
    Minneapolis, Minnesota 55455, United States
  • Washington University School of Medicine in St. Louis
    St Louis, Missouri 63110, United States
  • Hackensack University Medical Center
    Hackensack, New Jersey 07601, United States
  • Columbia University Irving Medical Center
    New York, New York 10032, United States
  • Memorial Sloan Kettering Cancer Center
    New York, New York 10065, United States
  • University Hospitals Cleveland Medical Center
    Cleveland, Ohio 44106, United States
  • The Cleveland Clinic Foundation
    Cleveland, Ohio 44195, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • Medical University of South Carolina
    Charleston, South Carolina 29425, United States
  • St Jude Children's Research Hospital
    Memphis, Tennessee 38105, United States
  • The Children's Hospital at TriStar Centennial
    Nashville, Tennessee 37203, United States
  • Medical College of Wisconsin
    Milwaukee, Wisconsin 53226, United States
07

References and documents

Publications

  • Gupta AO, Sharma A, Frangoul H, Kanter J, Mapara MY, Dalal J, Alavi A, Jaroscak JJ, Ayala E, DiPersio JF, Ziga ED, Eapen M, Rifkin-Zenenberg S, Minella AC, Chen Y, Chesler S, Ambati S, Bowman TS, Habtemariam B, Joseney-Antoine M, Chockalingam PS, Lin L, Goyal S, Simon A, Thompson AA, Heeney MM; BEACON Investigators. Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease. N Engl J Med. 2026 May 7;394(18):1824-1835. doi: 10.1056/NEJMoa2504835. Epub 2026 Apr 1. PubMed 41931046 ↗
  • 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. PubMed 38866240 ↗
  • 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. PubMed 36975201 ↗
  • 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. PubMed 36860093 ↗

Individual participant data

Plan to share: No

08

Registry details

Key details

Study ID
NCT05456880
Lead sponsor
Beam Therapeutics Inc.
Responsible party
Sponsor
First posted
Jul 13, 2022
Start date
Aug 30, 2022
Primary completion
Feb 2028 (estimated)
Completion
Feb 2028 (estimated)
Last update
Dec 16, 2025

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is active, not recruiting, as verified in Dec 2025. You cannot join it, but the record below documents what was studied.

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