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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05456880
Recruitment Status : Recruiting
First Posted : July 13, 2022
Last Update Posted : May 19, 2023
Sponsor:
Information provided by (Responsible Party):
Beam Therapeutics Inc.

Study Description
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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

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Biological: BEAM-101 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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) to Increase Fetal Hemoglobin (HbF) Production in Patients With Severe Sickle Cell Disease
Actual Study Start Date : August 30, 2022
Estimated Primary Completion Date : February 1, 2025
Estimated Study Completion Date : February 1, 2027

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Arms and Interventions
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Arm Intervention/treatment
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
Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 35 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria Include:

  1. Age ≥18 years to ≤35 years for the initial sentinel cohort; for subsequent enrollment patients from ≥12 years up to ≤35 years may be enrolled only upon approval by FDA.
  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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05456880


Contacts
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Contact: Medical Information 857-327-8641 clinicalinfo@beamtx.com

Locations
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United States, Florida
University of Miami Recruiting
Miami, Florida, United States, 33136-1005
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02215
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
United States, Tennessee
St Jude Children's Research Hospital Recruiting
Memphis, Tennessee, United States, 38105
United States, Wisconsin
Medical College of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Beam Therapeutics Inc.
More Information
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Responsible Party: Beam Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT05456880    
Other Study ID Numbers: BTX-AUT-001
First Posted: July 13, 2022    Key Record Dates
Last Update Posted: May 19, 2023
Last Verified: May 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Beam Therapeutics Inc.:
Gene Editing
Sickle Cell
Severe Sickle Cell
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn