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Clinical Trials in the USA / NCT05456880
Active, not recruiting Phase 1/2

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
No
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)

FacilityCityRegionStatus
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 Long-term Follow-up Study in Patients Who Received BEAM-101 Recruiting
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) Recruiting
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

Other trials for the same condition

A Long-term Follow-up Study in Patients Who Received BEAM-101 Recruiting
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy Enrolling by invitation
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease Active, not recruiting
A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS) Active, not recruiting
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) Active, not recruiting
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