Trials / Active Not Recruiting
Active Not RecruitingNCT05456880
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)
- Status
- Active Not Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 15 (estimated)
- Sponsor
- Beam Therapeutics Inc. · Industry
- Sex
- All
- Age
- 12 Years – 35 Years
- Healthy volunteers
- Not accepted
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
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | BEAM-101 | Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan |
Timeline
- Start date
- 2022-08-30
- Primary completion
- 2028-02-01
- Completion
- 2028-02-01
- First posted
- 2022-07-13
- Last updated
- 2025-12-16
Locations
19 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05456880. Inclusion in this directory is not an endorsement.