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Sickle Cell DiseasePhase 1/2BEACONNCT05456880

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

A Phase 1/2 study testing BEAM-101 in people with Sickle Cell Disease, run by Beam Therapeutics Inc. at 19 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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 Read more about this study

Who is it for?
Ages 12 to 35 years
Conditions the study is for
Sickle Cell Disease
What is being tested?
BEAM-101
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
19 sitesUnited States
Study dates
30 Aug 2022to Feb 2028
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 35 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

19 sites in 1 country.

19 sites in 1 country

United States19 sites

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

    Atlanta, Georgia, 30329

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35233

  • Boston Children's Hospital

    Boston, Massachusetts, 02215

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106

  • The Cleveland Clinic Foundation

    Cleveland, Ohio, 44195

  • Henry Ford Cancer Center

    Detroit, Michigan, 48202

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224

  • St Jude Children's Research Hospital

    Memphis, Tennessee, 38105

  • University of Miami

    Miami, Florida, 33136-1005

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • University of Minnesota

    Minneapolis, Minnesota, 55455

  • The Children's Hospital at TriStar Centennial

    Nashville, Tennessee, 37203

  • Columbia University Irving Medical Center

    New York, New York, 10032

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016

  • Washington University School of Medicine in St. Louis

    St Louis, Missouri, 63110

Other trials in United States

About this study

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)

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

Being tested
BEAM-101
Keywords
Gene Editing, Sickle Cell, Severe Sickle Cell
Medical subject headings
Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BEAM-101Biological

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

Study groups in NCT05456880
Study groupRoleWhat they receive
BEAM-101ExperimentalBEAM-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.
Receives: Biological: BEAM-101

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: 6 months to time of analysis as compared to baseline

  2. Proportion of patients with successful neutrophil engraftment

    Measured: BEAM-101 administration to month 24

  3. Time to neutrophil engraftment

    Measured: BEAM-101 administration to month 24

  4. Time to platelet engraftment

    Measured: BEAM-101 administration to month 24

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

    Measured: BEAM-101 administration to day 100

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

    Measured: BEAM-101 administration through month 24

Secondary outcomes (12)

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

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

  2. Proportion of patients experiencing no severe VOCs

    Measured: 6 months to time of analysis as compared to baseline

  3. Change in annualized number of hospitalizations for VOCs

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

  4. Change in annualized duration of hospitalizations for VOCs

    Measured: 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

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

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

    Measured: Baseline to month 24

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

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

  8. Change in lactate dehydrogenase (LDH) over time

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

  9. Change in total bilirubin over time

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

  10. Change in free Hgb over time

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

  11. Change in haptoglobin over time

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

  12. Change in reticulocyte count over time

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Jul 2022
  2. Study start30 Aug 2022
  3. Primary completionFeb 2028 (estimated)
  4. Study completionFeb 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
15 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05456880
Sponsor's protocol ID
BTX-AUT-001
Acronym
BEACON
Status last verified
Dec 2025
First submitted
23 Jun 2022
Last updated
16 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

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

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing BEAM-101 in people with Sickle Cell Disease, run by Beam Therapeutics Inc. at 19 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 16 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05456880, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Dec 2025.

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