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

NCT05456880 · tracked via the Priya Life Science USA tracker

Sponsor

Beam Therapeutics Inc.

Phase

Phase 1/2

Started

2022-08-30

Last updated

2025-12-16

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

BEAM-101

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

Study 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

Eligibility

Sex

ALL

Min age

12 Years

Max age

35 Years

Healthy volunteers

Key Inclusion Criteria Include: 1. Age ≥12 years to ≤35 years 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

Primary outcome measure(s)

Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline — 6 months to time of analysis as compared to baseline
Proportion of patients with successful neutrophil engraftment — BEAM-101 administration to month 24
Time to neutrophil engraftment — BEAM-101 administration to 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
Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's) — BEAM-101 administration through month 24

Trial sites (19)

Facility	City	Region
University of Alabama at Birmingham	Birmingham	Alabama
Phoenix Children's Hospital	Phoenix	Arizona
Mayo Clinic Florida	Jacksonville	Florida
University of Miami	Miami	Florida
Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital	Atlanta	Georgia
Boston Children's Hospital	Boston	Massachusetts
Henry Ford Cancer Center	Detroit	Michigan
University of Minnesota	Minneapolis	Minnesota
Washington University School of Medicine in St. Louis	St Louis	Missouri
Hackensack University Medical Center	Hackensack	New Jersey
Columbia University Irving Medical Center	New York	New York
Memorial Sloan Kettering Cancer Center	New York	New York
University Hospitals Cleveland Medical Center	Cleveland	Ohio
The Cleveland Clinic Foundation	Cleveland	Ohio
Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
Medical University of South Carolina	Charleston	South Carolina
St Jude Children's Research Hospital	Memphis	Tennessee
The Children's Hospital at TriStar Centennial	Nashville	Tennessee
Medical College of Wisconsin	Milwaukee	Wisconsin

More Beam Therapeutics Inc. trials in the USA

A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa) Recruiting

A Study Evaluating the Safety and Efficacy of BEAM-201 in Relapsed/Refractory T-Cell Acute Lymphoblastic Leukemia (T-ALL) or T-Cell Lymphoblastic Lymphoma (T-LL) Active, not recruiting

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) Recruiting

A Long-term Follow-up Study in Patients Who Received BEAM-101 Recruiting

Other trials for the same condition

A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease Recruiting

Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD) Recruiting

Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease Recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) Active, not recruiting

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy Enrolling by invitation

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05456880 on ClinicalTrials.gov ↗ ← All trials in the USA