ClinicalTrials.gg
Sign In
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

Browse trials for

Keywords

  • Gene Editing
  • Sickle Cell
  • Severe Sickle Cell
03

In context

Anemia, Sickle Cell

1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.

This study's planned enrollment of 15 is below the median of 40 across 769 interventional studies indexed under Anemia, Sickle Cell.

Browse Anemia, Sickle Cell studies →

Lead sponsor

Beam Therapeutics Inc. is the lead sponsor of 6 studies on the registry; 4 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

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 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
Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Allogeneic and autologous transplant

Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.

05

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

06

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

07

Study locations

19 sites
08

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

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Aug 2022 (actual). Primary completion is estimated for Feb 2028, 66 months after the start.

Of 37 phase 1/2 interventional studies under Anemia, Sickle Cell, half reached primary completion within 82 months of starting (the median), counting those still running; a quarter within 44 and three quarters within 222. This study's planned 66 months is below that.

How these figures are worked out, and which studies are left out of them.

Registered
Jun 23, 2022
First posted
Jul 13, 2022
StartActual
Aug 30, 2022
Primary completionEst.
Feb 2028
CompletionEst.
Feb 2028
Last update
Dec 16, 2025
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Dec 16, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

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 ↗

Not currently enrolling

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

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Anemia, Sickle Cell →

Other studies from this sponsor

Beam Therapeutics Inc.

Start the discussion