BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
NCT05456880 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 15
Last updated 2025-12-16
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
- BIOLOGICAL
-
BEAM-101
Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan
Sponsors & Collaborators
-
Beam Therapeutics Inc.
lead INDUSTRY
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 12 Years
- Max Age
- 35 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2022-08-30
- Primary Completion
- 2028-02-29
- Completion
- 2028-02-29
- FDA Drug
- Yes
Countries
- United States
Study Locations
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