Back to resultsBEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease (BEACON)
Primary Purpose
Sickle Cell Disease
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
BEAM-101
Sponsored by
About this trial
This is an interventional treatment trial for Sickle Cell Disease focused on measuring Gene Editing, Sickle Cell, Severe Sickle Cell
Eligibility Criteria
Key Inclusion Criteria Include:
- 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.
- 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
Sites / Locations
- University of MiamiRecruiting
- Boston Children's HospitalRecruiting
- University of MinnesotaRecruiting
- St Jude Children's Research HospitalRecruiting
- Medical College of WisconsinRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
BEAM-101
Arm Description
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.
Outcomes
Primary Outcome Measures
Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline
Proportion of patients with successful neutrophil engraftment
Time to neutrophil engraftment
Time to platelet engraftment
Transplant-related mortality within 100 days after beam-101 treatment
Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)
Secondary Outcome Measures
Proportion of patients experiencing at least 75% reduction in annualized rate of severe VOCs
Proportion of patients experiencing no severe VOCs
Change in annualized number of hospitalizations for VOCs
Change in annualized duration of hospitalizations for VOCs
Change in RBC transfusions per month and per year for SCD-related indications
Change in total Hgb (g/dL) concentration over time
Proportion of patients with HbF ≥30%, for at least 3 months
Change in lactate dehydrogenase (LDH) over time
Change in total bilirubin over time
Change in free Hgb over time
Change in haptoglobin over time
Change in reticulocyte count over time
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05456880
Brief Title
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
Acronym
BEACON
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
Study Type
Interventional
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 30, 2022 (Actual)
Primary Completion Date
February 1, 2025 (Anticipated)
Study Completion Date
February 1, 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Beam Therapeutics Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
Keywords
Gene Editing, Sickle Cell, Severe Sickle Cell
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
BEAM-101
Arm Type
Experimental
Arm Description
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 Type
Biological
Intervention Name(s)
BEAM-101
Intervention Description
Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan
Primary Outcome Measure Information:
Title
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
Title
Proportion of patients with successful neutrophil engraftment
Time Frame
BEAM-101 administration to month 24
Title
Time to neutrophil engraftment
Time Frame
BEAM-101 administration to month 24
Title
Time to platelet engraftment
Time Frame
BEAM-101 administration to month 24
Title
Transplant-related mortality within 100 days after beam-101 treatment
Time Frame
BEAM-101 administration to day 100
Title
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 Measure Information:
Title
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
Title
Proportion of patients experiencing no severe VOCs
Time Frame
6 months to time of analysis as compared to baseline
Title
Change in annualized number of hospitalizations for VOCs
Time Frame
Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Title
Change in annualized duration of hospitalizations for VOCs
Time Frame
Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Title
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
Title
Change in total Hgb (g/dL) concentration over time
Time Frame
Baseline to month 24
Title
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
Title
Change in lactate dehydrogenase (LDH) over time
Time Frame
Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Title
Change in total bilirubin over time
Time Frame
Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Title
Change in free Hgb over time
Time Frame
Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Title
Change in haptoglobin over time
Time Frame
Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Title
Change in reticulocyte count over time
Time Frame
Month 3 post BEAM-101 treatment to month 24 as compared to baseline
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria Include:
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.
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Medical Information
Phone
857-327-8641
Email
clinicalinfo@beamtx.com
Facility Information:
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136-1005
Country
United States
Individual Site Status
Recruiting
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Individual Site Status
Recruiting
Facility Name
St Jude Children's Research Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States
Individual Site Status
Recruiting
Facility Name
Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
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