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

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ClinicalTrials.gov Identifier: NCT05456880
Recruitment Status : Recruiting
First Posted : July 13, 2022
Last Update Posted : March 13, 2024
Sponsor:
Information provided by (Responsible Party):
Beam Therapeutics Inc.

Tracking Information
First Submitted Date  ICMJE June 23, 2022
First Posted Date  ICMJE July 13, 2022
Last Update Posted Date March 13, 2024
Actual Study Start Date  ICMJE August 30, 2022
Estimated Primary Completion Date February 1, 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 11, 2022)
  • 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 ]
  • Proportion of patients with successful neutrophil engraftment [ Time Frame: BEAM-101 administration to month 24 ]
  • Time to neutrophil engraftment [ Time Frame: BEAM-101 administration to month 24 ]
  • Time to platelet engraftment [ Time Frame: BEAM-101 administration to month 24 ]
  • Transplant-related mortality within 100 days after beam-101 treatment [ Time Frame: BEAM-101 administration to day 100 ]
  • Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's) [ Time Frame: BEAM-101 administration through month 24 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 11, 2022)
  • 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 ]
  • Proportion of patients experiencing no severe VOCs [ Time Frame: 6 months to time of analysis as compared to baseline ]
  • Change in annualized number of hospitalizations for VOCs [ Time Frame: Month 6 post BEAM-101 treatment to month 24 as compared to baseline ]
  • Change in annualized duration of hospitalizations for VOCs [ Time Frame: Month 6 post BEAM-101 treatment to month 24 as compared to baseline ]
  • 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 ]
  • Change in total Hgb (g/dL) concentration over time [ Time Frame: Baseline to month 24 ]
  • 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 ]
  • Change in lactate dehydrogenase (LDH) over time [ Time Frame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline ]
  • Change in total bilirubin over time [ Time Frame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline ]
  • Change in free Hgb over time [ Time Frame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline ]
  • Change in haptoglobin over time [ Time Frame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline ]
  • Change in reticulocyte count over time [ Time Frame: Month 3 post BEAM-101 treatment to month 24 as compared to baseline ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
Official Title  ICMJE 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
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
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE Biological: BEAM-101
Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan
Study Arms  ICMJE 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.
Intervention: Biological: BEAM-101
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 11, 2022)		 15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE February 1, 2027
Estimated Primary Completion Date February 1, 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 35 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Medical Information 857-327-8641 clinicalinfo@beamtx.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05456880
Other Study ID Numbers  ICMJE BTX-AUT-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Beam Therapeutics Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Beam Therapeutics Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Beam Therapeutics Inc.
Verification Date March 2024

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP