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NCT Number: NCT05456880

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

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

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

12 year–35 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

University of Alabama at Birmingham, Birmingham, Alabama, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria Include:

  • 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

Key Exclusion Criteria Include:

  • 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

Treatment and study plan

BEAM-101

Biological

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

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

Sponsors and collaborators

Lead sponsor

Beam Therapeutics Inc.

Industry

Registry information

Official study 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)

Acronym: BEACON

Important dates

Study start
2022
Primary completion
2028
Study completion
2028
First posted
Jul 13, 2022
Registry last updated
Dec 16, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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