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

Phase 1

Recruiting

• Conditions: Sickle Cell Disease
• Interventions: Biological: BEAM-101

• Registration Number: NCT05456880
• Lead Sponsor: Beam Therapeutics Inc.

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 available

Study Timeline

• Study First Submitted: 2022-06-23
• Study First Submitted QC: 2022-07-11
• Study First Posted: 2022-07-13
• Study Start: 2022-08-30
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-09
• Last Update Posted: 2025-01-10
• Primary Completion: 2025-02-01
• Study Completion: 2027-02-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
BEAM-101	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.

Primary Outcome Measures

Name	Time	Method
Proportion of patients with successful neutrophil engraftment	BEAM-101 administration to month 24
Time to neutrophil engraftment	BEAM-101 administration to month 24
Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)	BEAM-101 administration through month 24
Time to platelet engraftment	BEAM-101 administration to month 24
Transplant-related mortality within 100 days after beam-101 treatment	BEAM-101 administration to day 100
Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline	6 months to time of analysis as compared to baseline

Secondary Outcome Measures

Name	Time	Method
Change in free Hgb over time	Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Change in annualized duration of hospitalizations for VOCs	Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Proportion of patients with HbF ≥30%, for at least 3 months	Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Change in total bilirubin over time	Month 3 post BEAM-101 treatment to month 24 as compared to baseline
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
Proportion of patients experiencing no severe VOCs	6 months to time of analysis as compared to baseline
Change in lactate dehydrogenase (LDH) over time	Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Change in reticulocyte count over time	Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Change in total Hgb (g/dL) concentration over time	Baseline to month 24
Change in annualized number of hospitalizations for VOCs	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	Month 2 post BEAM-101 treatment to month 24 as compared to baseline
Change in haptoglobin over time	Month 3 post BEAM-101 treatment to month 24 as compared to baseline

Trial Locations

Locations (20)

Phoenix Children's Hospital; 🇺🇸 Phoenix, Arizona, United States

Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital; 🇺🇸 Atlanta, Georgia, United States

Hackensack University Medical Center; 🇺🇸 Hackensack, New Jersey, United States

Memorial Sloan Kettering Cancer Center; 🇺🇸 New York, New York, United States

The Cleveland Clinic Foundation; 🇺🇸 Cleveland, Ohio, United States

University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

Lucile Packard Children's Hospital at Standford; 🇺🇸 Palo Alto, California, United States

Mayo Clinic Florida; 🇺🇸 Jacksonville, Florida, United States

University of Miami; 🇺🇸 Miami, Florida, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Henry Ford Cancer Center; 🇺🇸 Detroit, Michigan, United States

University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States

Washington University School of Medicine in St. Louis; 🇺🇸 Saint Louis, Missouri, United States

Columbia University Irving Medical Center; 🇺🇸 New York, New York, United States

University Hospitals Cleveland Medical Center; 🇺🇸 Cleveland, Ohio, United States

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Medical University of South Carolina; 🇺🇸 Charleston, South Carolina, United States

St Jude Children's Research Hospital; 🇺🇸 Memphis, Tennessee, United States

The Children's Hospital at TriStar Centennial; 🇺🇸 Nashville, Tennessee, United States

Medical College of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States