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

Sickle Cell Disease Clinical Trial

— 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05456880
• Other study ID #: BTX-AUT-001
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: August 30, 2022
• Est. completion date: February 1, 2027

Study information

• Verified date: May 2024
• Source: Beam Therapeutics Inc.
• Contact: Medical Information
• Phone: 857-327-8641
• Email: clinicalinfo[@]beamtx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 15
• Est. completion date: February 1, 2027
• Est. primary completion date: February 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 35 Years

Eligibility

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 ßS/ßS, ßS/ß0, or ßS/ß+ 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

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

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

Locations

Country	Name	City	State
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Medical University of South Carolina	Charleston	South Carolina
United States	University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	Henry Ford Cancer Center	Detroit	Michigan
United States	Mayo Clinic Florida	Jacksonville	Florida
United States	St Jude Children's Research Hospital	Memphis	Tennessee
United States	University of Miami	Miami	Florida
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	University of Minnesota	Minneapolis	Minnesota
United States	The Children's Hospital at TriStar Centennial	Nashville	Tennessee
United States	Columbia University Irving Medical Center	New York	New York
United States	Lucile Packard Children's Hospital at Standford	Palo Alto	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Washington University School of Medicine in St. Louis	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Beam Therapeutics Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline		6 months to time of analysis as compared to baseline
Primary	Proportion of patients with successful neutrophil engraftment		BEAM-101 administration to month 24
Primary	Time to neutrophil engraftment		BEAM-101 administration to month 24
Primary	Time to platelet engraftment		BEAM-101 administration to month 24
Primary	Transplant-related mortality within 100 days after beam-101 treatment		BEAM-101 administration to day 100
Primary	Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)		BEAM-101 administration through month 24
Secondary	Proportion of patients experiencing at least 75% reduction in annualized rate of severe VOCs		Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Secondary	Proportion of patients experiencing no severe VOCs		6 months to time of analysis as compared to baseline
Secondary	Change in annualized number of hospitalizations for VOCs		Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Secondary	Change in annualized duration of hospitalizations for VOCs		Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Secondary	Change in RBC transfusions per month and per year for SCD-related indications		Month 2 post BEAM-101 treatment to month 24 as compared to baseline
Secondary	Change in total Hgb (g/dL) concentration over time		Baseline to month 24
Secondary	Proportion of patients with HbF =30%, for at least 3 months		Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Secondary	Change in lactate dehydrogenase (LDH) over time		Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Secondary	Change in total bilirubin over time		Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Secondary	Change in free Hgb over time		Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Secondary	Change in haptoglobin over time		Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Secondary	Change in reticulocyte count over time		Month 3 post BEAM-101 treatment to month 24 as compared to baseline