NCT05456880Active, not recruitingPhase 1/2
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients With Sickle Cell Disease and Severe Vaso-Occlusive Crises (Beacon Trial)
Source: ClinicalTrials.gov · Last updated 16 Dec 2025
Brief summary (registry, in English)
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
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 15 (estimated)
- First submitted
- 23 Jun 2022
- Start date
- 30 Aug 2022
- Primary completion date
- Feb 2028
- Lead sponsor
- Beam Therapeutics Inc.
- Diseases
- Sickle cell disease
- NCT number
- NCT05456880
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 15
- From 30 Aug 2022 to the primary completion date, Feb 2028
- 19 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
Other illness
- Definitive diagnosis of moyamoya syndrome based on screening brain MRA
- History of overt stroke
Other
- Available and willing matched sibling donor
Who the study is looking for
- Age ≥12 years to ≤35 years
- 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
Age in the registry: 12 years to 35 years · Sex: All · Does not accept healthy volunteers
Sites (19)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital Coordinator of this site? Confirm its status | Atlanta | United States | Unknown status |
| University of Alabama at Birmingham Coordinator of this site? Confirm its status | Birmingham | United States | Unknown status |
| Boston Children's Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Medical University of South Carolina Coordinator of this site? Confirm its status | Charleston | United States | Unknown status |
| University Hospitals Cleveland Medical Center* Coordinator of this site? Confirm its status | Cleveland | United States | Unknown status |
| The Cleveland Clinic Foundation Coordinator of this site? Confirm its status | Cleveland | United States | Unknown status |
| Henry Ford Cancer Center Coordinator of this site? Confirm its status | Detroit | United States | Unknown status |
| Hackensack University Medical Center Coordinator of this site? Confirm its status | Hackensack | United States | Unknown status |
| Mayo Clinic Florida Coordinator of this site? Confirm its status | Jacksonville | United States | Unknown status |
| St. Jude Children's Research Hospital Coordinator of this site? Confirm its status | Memphis | United States | Unknown status |
| University of Miami Coordinator of this site? Confirm its status | Miami | United States | Unknown status |
| Medical College of Wisconsin Coordinator of this site? Confirm its status | Milwaukee | United States | Unknown status |
| University of Minnesota Coordinator of this site? Confirm its status | Minneapolis | United States | Unknown status |
| The Children's Hospital at TriStar Centennial Coordinator of this site? Confirm its status | Nashville | United States | Unknown status |
| Memorial Sloan-Kettering Cancer Center Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Columbia University Irving Medical Center Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Children's Hospital of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Phoenix Children s Hospital Coordinator of this site? Confirm its status | Phoenix | United States | Unknown status |
| Washington University School of Medicine in St. Louis Coordinator of this site? Confirm its status | St Louis | United States | Unknown status |
Related publications
- Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease.N Engl J Med · 2026 · PMID 41931046
This page is information, not medical advice. Discuss any trial with your doctor.