NCT07373639

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

A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 50 (estimated)
• Sponsor: Beam Therapeutics Inc. · Industry
• Sex: All
• Age: 14 Years – 37 Years
• Healthy volunteers: Not accepted

Summary

This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing Study BTX-AUT-001. Patients who enter into this study will be followed for 13 years (a total of 15 years after receiving BEAM-101).

Detailed description

Patients who were treated with BEAM-101 in Study BTX-AUT-001 will be asked to sign an informed consent form (ICF) to enter this LTFU study (during the EOS/Month 24 visit for BTX-AUT-001). Patients in this study will have periodic safety and efficacy assessments per the schedule of assessments (SoA). Visits will occur annually through Year 5 following BEAM-101 administration and then every 3 years through Year 11, with a final visit at Year 15. Virtual/phone call check-in visits will occur every 6 months through Year 5 and then annually thereafter.

Conditions

• Hemoglobinopathy
• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Long-Term Follow-up Study of patients who received BEAM-101	This is a Long-Term Follow-up Study of patients who received a single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan

Timeline

• Start date: 2025-12-15
• Primary completion: 2043-02-12
• Completion: 2043-02-12
• First posted: 2026-01-28
• Last updated: 2026-01-28

Locations

17 sites across 1 country: United States

Regulatory

• FDA-regulated drug study